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CALL FOR PROPOSALS 2012 INSTRUCTIONS FOR PREPARATION OF APPLICATIONS Before completing an application, you must consult these “Instructions for Preparation of Applications” and follow the directions. CONTENTS 1. Forms (applications, submissions, types of financing) 2. Information relative to the evaluation of a project and its contractual engagements 3. Information about financing 4. Specific cases 5. Preparation of the application and list of documents to include 6. Criteria for evaluation of applications 7. Annex: Letter of Agreement, in French and in English 1. Forms (applications, submissions, types of financing) APPLICATIONS: The application forms (research project, research project renewal, trampoline grant, PhD or postdoctoral fellowship, PhD or postdoctoral fellowship renewals) are revised each year. Before completing an application it is imperative that you verify that you have the current version (2012), which is available on the AFM Website, or you will risk inadmissibility of the application. SUBMISSION OF APPLICATIONS: APPLICATIONS MUST BE IMPERATIVELY ADDRESSED: 1/ By e-mail, to: [email protected] and 2/ By regular mail (! ATTENTION, address has changed), to: Mme Claire LEGRAND-TABARY - 1 - ASSOCIATION FRANCAISE CONTRE LES MYOPATHIES SPCS / Permanent Secretariat of the Scientific Council 96, rue Didot - 75014 PARIS France Tel : 33 1 40 52 07 66 - Fax : 33 1 40 52 05 75

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CALL FOR PROPOSALS 2012

INSTRUCTIONS FOR PREPARATION OF APPLICATIONS

Before completing an application, you must consult these “Instructions for Preparation of Applications” and follow the directions.

CONTENTS

1. Forms (applications, submissions, types of financing)

2. Information relative to the evaluation of a project and its contractual engagements

3. Information about financing

4. Specific cases

5. Preparation of the application and list of documents to include

6. Criteria for evaluation of applications

7. Annex: Letter of Agreement, in French and in English

1. Forms (applications, submissions, types of financing)

APPLICATIONS:

The application forms (research project, research project renewal, trampoline grant, PhD or postdoctoral fellowship, PhD or postdoctoral fellowship renewals) are revised each year. Before completing an applica-tion it is imperative that you verify that you have the current version (2012), which is available on the AFM Website, or you will risk inadmissibility of the application.

SUBMISSION OF APPLICATIONS:

APPLICATIONS MUST BE IMPERATIVELY ADDRESSED:1/ By e-mail, to: [email protected] and2/ By regular mail (! ATTENTION, address has changed), to:

Mme Claire LEGRAND-TABARYResponsable du Secrétariat Permanent du Conseil ScientifiqueA.F.M. / SPCS 96, rue DIDOT75014 PARISFrancePhone number: 33 1 40 52 05 71 – Fax number: 33 1 40 52 05 75For more information, please contact: [email protected]

The applications must be sent, at the latest (according to the postmark):- October 7, 2011 for the first session- March 9, 2012 for the second session

- 1 -ASSOCIATION FRANCAISE CONTRE LES MYOPATHIES

SPCS / Permanent Secretariat of the Scientific Council96, rue Didot - 75014 PARIS France

Tel : 33 1 40 52 07 66 - Fax : 33 1 40 52 05 75

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TYPES OF FINANCING:

Research projects for one year, renewable for a second year, and exceptionally for a third year. Trampoline grants: This one-year award is intended to support investigators early in their professional ca-reer who will propose innovative projects. This grant is awarded for a maximum of 50,000 euros for one year. PhD fellowships for 1 year, renewable for a second and third year. Postdoctoral fellowships for 1 year, renewable for a second year.Ambitious research projects with PhD or post-doctoral fellowships will be recognised as an AFM-sponsored programs.

2. Information relative to the evaluation of a project and its contractual engagementsa. Your application for financing will undergo a procedure of a priori scientific expertise (submission of your project to external experts and to members of the Scientific Council of AFM). After this phase, your project will be submitted to the Board of Directors of AFM, who will either refuse or accept to finance your project.

b. You could be informed of the decision of the Board of Directors of AFM.

For Deadline October 7, 2011, decisions are expected mid-February 2012For Deadline March 9, 2012, decisions are expected mid-July 2012.

As a general rule, and with the exception of Fellowships, a Letter of Agreement will accompany acceptance letters, based on the model included as an annex to the present document, which must imperatively be signed by the legal representative of your organization (i.e. your employer or his representative) before any disbursement of funds by AFM. Very exceptionally, AFM may wish, based on certain criteria (e.g. major strategic importance of your project for AFM, notably in terms of development of therapeutics, amount of AFM financing, etc.), to manage your fin-ancing as a contract negotiated and signed by AFM and your organization. In this contract, in return for the fin-ancing granted for your project, AFM will notably request the granting of intellectual property rights on the res-ults of the project, as well as return on investment if results of the project generate exploitation revenues.

c. We would like to draw your attention to the duration of this procedure and the fact that no disbursement of funds will be made until AFM receives this Letter of Agreement duly signed by the legal representative of your organization.

d. To expedite this process and shorten the time frame for signature of the above-mentioned documents, we strongly encourage you to inform and transmit the following documents to your employer or his representative (e.g. Technology Transfer, Intellectual Property, Patents, Partnership, Legal departments) as soon as possible:

A copy of your Application for Financing submitted to AFM A copy of the “Instructions for preparation of applications”

It is not necessary to send us the "Lettre d'Engagement" (in annex) at this time, since this has no legal value at the moment.

3. Information about financing’s request (For additional information about financing’s request, you can contact Catherine Hivart, 33.1.69.13.21.48, [email protected])

Direct project costs: Direct project costs should be related to the realization of the project for the duration of the project.The amounts should be in euros.

If the taxes related to the costs of the projects are recoverable by the organization financed, the amounts should be indicated without tax; if not, indicate the amounts including taxes.

Definitions for the number of person-months and salaries:

- 2 -ASSOCIATION FRANCAISE CONTRE LES MYOPATHIES

SPCS / Permanent Secretariat of the Scientific Council96, rue Didot - 75014 PARIS France

Tel : 33 1 40 52 07 66 - Fax : 33 1 40 52 05 75

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*1 person-month (PM) corresponds to the work performed by one full-time person for one month

Examples:12 PM = 1 person working full-time for 1 year12 PM = 1 person working 50% of full-time (half time) for 2 years12 PM = 2 persons working half-time for 1 year

Salary: gross salary/employer contributionsContributions (unemployment, pension, health insurance, etc.) of the employer are calculated on the basis of the gross salary.

Salary cost: gross salary + employer contributionsThe total cost paid by the enterprise is the salary cost.

The date when the research project is considered to begin is the date of signature of the Letter of Agreement.

Precisions concerning remunerations for PhD and post-doctoral fellowships:

FRANCE (SALARIES) FOREIGN COUNTRIES (LIBERALITIES)PhD fellowships 1684,73 euros/month

(all taxes included)16 500 euros/year

Post-Doctoral fellow-ships

From 2500 to 3319 euros/month (all taxes included)

25.500 euros/year

4. Specific casesREFORMULATION AND REVISED APPLICATIONS OF A PROJECT

If your request is a reformulation or a revision of a preceding project which was not accepted by AFM, append a point-by-point response to the remarks and questions of the experts (in English) - you need not agree with all points, but you must address them. Use page numbers and other identifiers so reviewers can easily find where you have added new data or revised experimental approaches. A bar in the margin is a good way to show where revisions are.  Highlight new sections with indenting, bracketing, underlining, or change of type. If you disagree with the reviewers, explain why and provide additional information if needed. An angry rebuttal letter is un-likely to be well-received, even by a new committee member. Maintain as positive a tone as possible.

Please note that even if you respond adequately to the criticisms, you are not guaranteed an award. This may happen because a summary statement is not meant to be an exhaustive critique; some problems discussed by the reviewers may not appear in it. Instead, it hits the highlights of the review discussion, providing general feed-back. Also, when you make changes, you risk introducing new problems. Finally, membership in scientific re -view groups changes. Your application may be seen by some new reviewers who may have different views of your project.

This additional document should be sent 1/ as one paper copy appended to the application, and 2/ as one elec-tronic version at the same time as you send the electronic version of your application.

PROJECT INCLUDING MORE THAN 1 TEAM

For applications for financing which include at least 2 groups (collaborative network project): the application should be completed in the following manner:

The request should be sent in the name of the coordinator of the network; the co-ordinates of this per -son should be indicated on page 1

The summary (in English and in French if possible) should mention each of the teams implicated, as well as the name of the applicant representing each of the teams.

The description of the research project should include a section explaining the interactions and com -plementarity between the groups. The investigator from each team should explain his own role in the research project.

The participants in the programme (page 12) should be listed team by team.

5. Preparation of the application and list of documents to include.- 3 -

ASSOCIATION FRANCAISE CONTRE LES MYOPATHIESSPCS / Permanent Secretariat of the Scientific Council

96, rue Didot - 75014 PARIS FranceTel : 33 1 40 52 07 66 - Fax : 33 1 40 52 05 75

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ELIGIBILITY FOR THE VARIOUS TYPES OF FINANCING (If these conditions are not fulfilled, your applica-tion will be automatically rejected):

Application for research project: In the academic sector, only tenured faculty may apply. Application for research project renewal: In the academic sector, only tenured faculty may apply. You have previously applied to AFM for a research project for a duration of 2 years on the same topic, and your ac -ceptance letter mentioned financing for a second year subject to submission of a renewal application before one of the deadlines announced in our Call for Proposals. Application for Trampoline grant: This one-year award is intended to support investigators early in their professional career who will propose innovative projects. Please note that aside from presenting outstanding science, a Trampoline grant application should be “high risk/high yield”, i.e. does not need preliminary data but the idea should be extremely innovative and if correct has the potential of being groundbreaking. This grant is in no way a mean to allude the more demanding regular research program.    Application for PhD fellowship: Master’s degree (M2 in France) or equivalent must be acquired. Please note that it is mandatory for students who have been supported by AFM for their thesis work to participate in an annual « Young Researchers forum » organized by AFM. An unjustified absence from this “Young Researchers forum” will lead to an unfavorable recommendation in case of application for renewal. Application for PhD fellowship renewal: Candidates who have previously received PhD support from AFM for the same subject in the same laboratory as in this application. The application should be submitted within the 6 months following the end of AFM support at the latest; any extension of this deadline must be justified in a letter accompanying this application. Please note that it is mandatory for students who have been suppor-ted by AFM for their thesis work to participate in an annual « Young Researchers forum » organized by AFM. An unjustified absence from this “Young Researchers forum” will lead to an unfavorable recom-mendation in case of application for renewal. Application for Postdoctoral fellowship: Be in possession of a PhD degree on the date when the financial support is due to start. Postdoctoral candidates must demonstrate mobility in their career path. Any exceptions to this rule must be clearly argued, and the duration of support will be reduced to one year. Application for Postdoctoral fellowship renewal: Candidates who have previously received postdoctoral support from AFM for the same subject in the same laboratory as in this application. The application should be submitted within the 6 months following the end of AFM support at the latest; any extension of this deadline must be justified in a letter accompanying this application.

FOR ALL TYPES OF APPLICATIONS:

BE CAREFUL THAT APPLICATIONS LEFT IN THE CORRECTIVE MODE ON PAPER OR ELEC-TRONIC VERSIONS WILL BE SYSTEMATICALLY REFUSED.

The “original” document will include original signatures and stamps. Recto-verso documents are preferable. No cover page should be placed before page 1 of the application form. For the electronic version of the complete application (to be sent by e-mail): We will no longer accept more than 4 electronic files per application; each file should not exceed 2.0 Mb. Your files should be named in the fol-lowing manner:

For the application form: FAMILY NAME-First name-Project For the “synthesis” of the application (pages 1 to 6): FAMILY NAME – First name - synthesis For any annexes (figures or other): FAMILY NAME -First name-complementary information For the CV (only for Fellowships): FAMILY NAME-First name-CV. Please DO NOT include date

of birth, age, place of birth, citizenship, marital status, sex, religious affiliation and do not use photograph.

FOR RESEARCH PROJECTS OR TRAMPOLINE GRANTS:

1 signed original application form. The signatures of the Laboratory Director (page 1) and of all the participants (page 12) must appear on this application.

- 4 -ASSOCIATION FRANCAISE CONTRE LES MYOPATHIES

SPCS / Permanent Secretariat of the Scientific Council96, rue Didot - 75014 PARIS France

Tel : 33 1 40 52 07 66 - Fax : 33 1 40 52 05 75

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1 electronic version of the pages 1-6 (“Table of request for financing” included) necessarily as a Word file (to be sent by e-mail to [email protected]).

1 electronic version of the complete application (to be sent by e-mail to [email protected])

FOR RESEARCH PROJECTS RENEWAL:

1 signed original application form. The signatures of the Laboratory Director (page 1) and the signa-tures of all the participants (page 5) must appear on this application

1 electronic version of the pages 1-6 (“Budget review” included) necessarily as a Word file (to be sent by e-mail to [email protected]).

1 electronic version of the complete application (to be sent by e-mail to [email protected])

FOR FELLOWSHIPS (PhD, PhD renewal, Postdoctoral, or Postdoctoral renewal):

1 signed original application form including your CV in English (2 pages maximum). The signa-ture of the Laboratory Director (page 1) must appear on this application.

1 electronic version of the pages 1-6 (“Abstract in English” included), followed by your CV in English, necessarily as a Word file (to be sent by e-mail to [email protected]).

1 electronic version of the complete application (to be sent by e-mail to [email protected])

INCOMPLETE APPLICATIONS WILL NOT BE CONSIDERED

6. Criteria for application evaluation Each project will be evaluated according to the following criteria:

1. Relevance and significance for AFM 2. Project quality and  scientific soundness; 3. Innovation; 4. Feasibility (i.e., Investigator, environment, approach); 5. Economic reasonableness of the proposed budget;

1. Relevance and significance for AFM: How is this research relevant to a better understanding of neuromus-cular or other rare diseases, their causes, cures and/or prevention? Does this study address an important prob-lem?  Is there a sufficient body of high quality fundamental, preclinical or clinical research evidence that sup -ports the rationale for the proposed study?  What is the potential impact of the proposed intervention on health care and quality of life?2. Project quality/ scientific soundness:  Are the rationale and experimental design adapted to the study object-ives?3- Innovation:  Are the aims of the study original?  Does the proposed study design represent advancement in the field? 4. Feasibility:  Are the conceptual framework, design, methods, and analyses adequately developed, well integ-rated, and appropriate for the aims of the project?  Does the applicant acknowledge potential problem areas and consider alternative strategies? Is the principal investigator well suited to carry out the proposed study? Is the scientific environment adequate? Is there evidence of institutional support? Have appropriate agreements with collaborators been established?5. Reasonableness of the budget:  Is the budget reasonable in relation to the objectives of the study?

Notification: All candidates will be notified by mail on the decision made by the AFM board of Directors based on the re-commendations by the scientific council. Please note that the selection process is very competitive and might result in your application not being funded despite positive reviews . Notifications will be sent until Mid-April 2012 for the first session applications, and until Mid-September 2012 for the second session applica-tions. If you want to be informed before these dates, please contact us either by phone or by email.

7. Letter of Agreement, in French and in English

- 5 -ASSOCIATION FRANCAISE CONTRE LES MYOPATHIES

SPCS / Permanent Secretariat of the Scientific Council96, rue Didot - 75014 PARIS France

Tel : 33 1 40 52 07 66 - Fax : 33 1 40 52 05 75

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LETTRE D'ENGAGEMENT

Conformément à la décision de son Bureau du Conseil d’Administration du [DATE] , l’Association Française contre les Myopathies, association régie par la loi du 1er juillet 1901, reconnue d'utilité publique, ayant son siège social à 47-83, boulevard de l'Hôpital, 75651 Paris cedex 13 (ci-après désignée l’« AFM »), s’apprête à financer le programme de recherche intitulé "«[DOSSIER]Titre_du_projet»" et référencé sous le numéro «[DOSSIER]Nro_AFM», ci-après désigné le « Programme », dirigé par le Dr./Pr. [DEMANDEUR], ci-après désigné le « Demandeur ». Le laboratoire dans lequel le Dr./Pr. [DEMANDEUR] réalise le Programme, dépend de [ORGANISME], ci-après désigné l’ « Organisme ».

Pour les besoins de financement de ce Programme, l’Organisme et l’AFM, ci-après collectivement désignés les « Parties », ou individuellement par la « Partie », conviennent expressément par les présentes ce qui suit :

1- LE FINANCEMENT DE L’AFM VISE PAR LES PRESENTES EST SUBORDONNE A LA SIGNATURE DE CETTE LETTRE D’ENGAGEMENT.

2- Financement accordé par l’AFML’Organisme et le Demandeur s’engagent à affecter exclusivement le financement accordé par l’AFM à l’exécution du Programme, et à aucune autre fin ou moyen.

Le versement du financement de l’AFM correspondant à cent pour cent (100%) du financement accordé par l’AFM au titre de la première année du Programme, sera réglé auprès de l’Organisme dans un délai de  soixante (60) jours ouvrés suivant la date de signature des présentes.

Dans l’hypothèse où (i) l’Organisme et le Demandeur, auraient initialement fait une demande de financement sur deux (2) ans et, (ii) ils auraient déposé dans les dix-huit (18) mois suivant la signature des présentes le Rapport d’Activités (selon la procédure prévue à l’Article 6 des présentes « Rapport(s) d’activités ») correspondant à la première année du Programme et, (iii) le Conseil d’Administration de l’AFM approuverait sur la base de ce Rapport d’Activités un renouvellement du financement du Programme pour une seconde année, un (1) versement supplémentaire, correspondant à cent pour cent (100%) du financement accordé par l’AFM au titre de la seconde année du Programme, pourrait être réglé auprès de l’Organisme dans un délai de soixante (60) jours ouvrés suivant la date de signature de la Lettre d’engagement relative à la seconde année. 

3- ConfidentialitéChaque Partie s'engage à traiter et à conserver de manière confidentielle toutes les informations confidentielles appartenant à l’autre Partie qu’elle aurait reçues de cette dernière à l’occasion de l’exécution du Programme pendant une durée de dix (10) ans à compter de la signature des présentes.L'AFM s'engage à traiter et à conserver de manière confidentielle toutes les informations qui lui sont transmises par le Demandeur aux fins d'évaluation par les membres de son Conseil Scientifique, les experts extérieurs sollicités ponctuellement (Demande de financement, Rapport d'activités), et par son Service compétent en matière de Propriété Industrielle. 4- Propriété et Valorisation industrielle des résultatsLe Programme de recherche financé par l'AFM a principalement un but expérimental. Il est néanmoins convenu que les résultats obtenus dans le cadre du Programme peuvent avoir une valeur commerciale et générer des droits de Propriété Intellectuelle. L’Organisme est propriétaire des résultats du Programme.Si les résultats obtenus sont susceptibles de protection par un droit de propriété intellectuelle, l’Organisme et le Demandeur s’engagent à les transmettre à la Direction Scientifique de l'AFM pour information.Si l’Organisme:

- ne souhaite pas protéger les résultats du Programme par un titre de propriété indus-trielle, ou

- souhaite abandonner un titre de propriété industrielle portant sur tout ou partie des ré-sultats du Programme,

- 6 -ASSOCIATION FRANCAISE CONTRE LES MYOPATHIES

SPCS / Permanent Secretariat of the Scientific Council96, rue Didot - 75014 PARIS France

Tel : 33 1 40 52 07 66 - Fax : 33 1 40 52 05 75

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l’Organisme pourra en informer l’AFM afin que cette dernière ait la possibilité d’assurer la protection des résultats du Programme en son nom et pour son propre compte.L’Organisme, et le cas échéant l’AFM, feront leurs meilleurs efforts pour évaluer les possibilités de protection et d’exploitation des résultats du Programme, sans retarder, le cas échéant, le dépôt d'une demande de brevet et/ou sans bloquer la publication desdits résultats dans des revues scientifiques.5- PublicationsLe Demandeur et l’Organisme s'engagent à faire figurer le nom de l'AFM dans les remerciements qui suivent généralement le texte des publications. Toute publication qui présentera le nom de l'AFM dans ses remerciements sera adressée dans les meilleurs délais à la Direction Scientifique de l'AFM et le pdf de la publication devra être envoyé à [email protected].

L’Organisme et le Demandeur autorisent expressément par la présente l’AFM à faire usage, dans le cadre de communications relatives au Programme, des noms de [l’ORGANISME DE RATTACHEMENT] et du [DEMANDEUR], du montant du financement accordé par l’AFM, du titre du Programme, du résumé non confidentiel du Programme en anglais de 5 lignes transmis par le Demandeur dans la demande de financement (lay summary), ainsi qu’à faire état du Programme oralement et/ou par écrit, au Grand Public notamment pendant ses campagnes de collecte de dons, pendant le Téléthon et l’Assemblée Générale de l’AFM, sans détails susceptibles de nuire aux droits de propriété intellectuelle issus du Programme.6– Rapport(s) d'activitésLe Rapport d’Activités se définit comme suit : il comporte un volet scientifique et un volet financier et doit respecter le modèle de rapport figurant en Annexe 1 des présentes. Le volet scientifique doit décrire les activités scientifiques réalisées en rapport avec les objectifs prévus dans le Programme, l’ensemble des résultats obtenus pendant l’année écoulée ainsi que les perspectives pour l’année suivante (si demande de renouvellement du financement). Le volet financier doit comporter (i) un bilan de l’utilisation du financement accordé par l’AFM pour l’année écoulée (ce bilan financier devra mettre en évidence, le cas échéant, le montant du financement non dépensé au cours de la période considérée, susceptible d’être soit restitué à l’AFM, soit, le cas échéant, reporté suivant décision du Conseil d’Administration de l’AFM à une seconde année du Programme) et (ii) un budget prévisionnel pour l’année suivante (si demande de renouvellement du financement).

Pour les programmes faisant l’objet d’une demande de renouvellement :Dans l’hypothèse où l’AFM aurait accepté le principe d’un financement du Programme sur deux (2) ans, l’Organisme et le Demandeur s’engagent à intégrer le Rapport d’Activités (incorporant un bilan financier intermédiaire) se rapportant à la première année du Programme dans une demande de renouvellement lors d’une session de l’appel d’offres de l’AFM et ce, dans un délai maximal de dix-huit (18) mois à compter de la date de signature de cette Lettre d’Engagement (pour plus de renseignements sur la procédure à respecter, le Demandeur peut s’adresser à [email protected]). Par ailleurs, dans un délai maximal de dix-huit (18) mois à compter de la date de signature de la Lettre d’Engagement relative à la seconde année du Programme, l’Organisme et le Demandeur s’engagent à fournir à l’AFM un Rapport final d’Activités (incorporant un bilan financier final couvrant la durée totale du Programme), tel que défini ci-dessus.

Pour les programmes ne faisant pas l’objet d’une demande de renouvellement :Dans l’hypothèse où l’AFM ne s’engagerait à financer qu’une (1) seule année du Programme, l’Organisme et le Demandeur s’engagent à fournir au Secrétariat Permanent du Conseil Scientifique de l'AFM, un Rapport d'Activités dans un délai maximal de dix-huit (18) mois à compter de la date de signature de cette Lettre d’Engagement.

7- Durée de l'engagementLa présente Lettre d’Engagement produira ses effets à compter de la date de sa signature et ce pendant une durée de dix-huit (18) mois. Les dépenses peuvent être encourues par l’Organisme pendant la durée de la présente Lettre d’Engagement, soit une durée de dix-huit (18) mois à compter de sa signature.

- 7 -ASSOCIATION FRANCAISE CONTRE LES MYOPATHIES

SPCS / Permanent Secretariat of the Scientific Council96, rue Didot - 75014 PARIS France

Tel : 33 1 40 52 07 66 - Fax : 33 1 40 52 05 75

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A l’échéance de la présente Lettre d’Engagement, les Articles 3, 4, 5 et 6 continueront à s’appliquer.

L’Annexe 1 « Modèle de Rapport d’Activités » fait partie intégrante de la présente Lettre d’Engagement.

Fait à Paris, le _____________

Le Demandeur Le Directeur du LaboratoireNom : Nom : Signature précédée de la mention Signature précédée de la mention"lu et approuvé" "lu et approuvé"

Le Représentant légal de l’OrganismeNom : Signature précédée de la mention :"Reconnaissant être dûment habilité à représenter l’Organisme aux fins des présentes, lu et approuvé"

- 8 -ASSOCIATION FRANCAISE CONTRE LES MYOPATHIES

SPCS / Permanent Secretariat of the Scientific Council96, rue Didot - 75014 PARIS France

Tel : 33 1 40 52 07 66 - Fax : 33 1 40 52 05 75

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Annexe 1Modèle de Rapport d’Activités

Scientific progress reporta) Describe the main goal and specific aims as described in the original application

b) Progress report: Describe in detail the accomplishments including all results, challenges, setbacks and explain, if applicable, the rationale for new directions taken. If the Project is a clinical trial, describe the progress of the trial in detail (regulatory, patients enrolment, serious adverse events, …). At the end of the trial, send a copy of the final clinical report written for regulatory authorities.

c) Quantify progress made towards the initial aims

d) Next steps

e) Publications and /or Patents

List the publications acknowledging the financial contribution of AFM including papers submitted for publication, accepted for publication or in press (References must in-clude names of all authors, full title of the manuscript, name of the journal the manu-script has been submitted to and date of submission). Please send a copy of the accep-ted papers to [email protected].

Intellectual property and patent(s) applicationThe applicant shall promptly notify AFM in writing of each Result and any patent applications filed thereon resulting from the research Project. (“Result” means any product, process, result, computer software, database, idea, information, development, conceived or created by the applicant and his/her program team, arising out of the research Project funded by AFM, that is or may be patentable or otherwise protectable by intellectual property rights).

- 9 -ASSOCIATION FRANCAISE CONTRE LES MYOPATHIES

SPCS / Permanent Secretariat of the Scientific Council96, rue Didot - 75014 PARIS France

Tel : 33 1 40 52 07 66 - Fax : 33 1 40 52 05 75

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- 10 -ASSOCIATION FRANCAISE CONTRE LES MYOPATHIES

SPCS / Permanent Secretariat of the Scientific Council96, rue Didot - 75014 PARIS France

Tel : 33 1 40 52 07 66 - Fax : 33 1 40 52 05 75

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LETTER OF AGREEMENT

In accordance with the decision of its Board of Directors on [DATE], the Association Française contre les Myopathies, governed by the Law of July 1, 1901, recognized as a public service organization, with its headquarters at 47-83 Boulevard de l’Hopital, 75651 Paris cedex 13 (hereinafter designated “AFM”) intends to finance the research program entitled "«[DOSSIER]Titre_du_projet»", AFM reference number DOSSIER]No_AFM», hereinafter designated the “Program”, directed by Dr./Prof [DEMANDEUR], hereinafter designated the “Applicant”. Dr./Prof. [DEMANDEUR] is affiliated with the [ORGANISME], hereinafter designated the “Institution” where the Programme will be carried out.

For the purposes of financing this Program, the Institution and AFM, hereinafter collectively designated as the “Parties”, or individually as the “Party”, agree expressly by the present Letter of Agreement that:

1. AFM’S FUNDING AS DESCRIBED IN THE PRESENT DOCUMENT IS SUBJECT TO SIGNATURE OF THIS LETTER OF AGREEMENT

2. AFM’s fundingThe Institution and the Applicant agree to allocate AFM’s funding exclusively for the execution of the Program, and not for any other purpose or means. The payment of AFM funding corresponding to one hundred percent (100%) of the financing allocated by AFM for the first year of the Program, will be sent to the Institution within sixty (60) working days following the date of signature of the present Letter of Agreement.

If (i) the Institution and the Applicant have made an initial funding request for a two (2)-year period, and if (ii) they have provided AFM with a report for the first year’s execution of the Program (in accordance with the procedure mentioned in the Article 6 “Progress Report(s)”), within eighteen (18) months following the date of signature of the present Letter of Agreement, and if (iii) AFM’s Board of Directors approves on the basis of this Progress Report, the continuation of the funding for a second year, one (1) supplementary payment corresponding to 100 percent (100%) of the financing allocated by AFM for the second year of the Program will be paid to the Institution within sixty (60) working days following the date of signature of the Letter of Agreement relative to the second year.

3. ConfidentialityEach Party agrees to keep and treat all information belonging to the other Party that is received during the execution of the Program in a confidential manner for a period of ten (10) years starting on the date of signature of the present document.AFM agrees to treat and keep confidential all information divulged to it by the Applicant for the purposes of evaluation by members of its Scientific Council, by selectively solicited external experts (Call for Proposals, Progress Report), and by its department which is competent in matters of industrial property.

4. Intellectual property and Industrial valorisation of the results.The research Program funded by AFM has a principally experimental goal. It is nevertheless acknowledged that the results obtained in the context of the Program may have a commercial value and generate Intellectual Property rights. The Institution is the owner of the results of the Program.If the results obtained are subject to protection by any intellectual property right, the Institution and the Applicant agree to transmit them to the Scientific Department of AFM for information.If the Institution

- does not wish to protect the results of the Program by industrial property rights, or - wishes to abandon industrial property rights on all or part of the results of the Program,

the Institution may inform AFM of this so that AFM has the possibility to ensure protection of the results of the Program in its name and for its own account.The Institution and, if need be, AFM, will provide their best efforts to evaluate the possibilities of protection and exploitation of the results of the Program without delaying, in such cases, the filing of a patent and/or without blocking publication of the said results in scientific journals.

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SPCS / Permanent Secretariat of the Scientific Council96, rue Didot - 75014 PARIS France

Tel : 33 1 40 52 07 66 - Fax : 33 1 40 52 05 75

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5. PublicationsThe Applicant and the Institution agree to place the name of AFM in the acknowledgements section which generally follows the text in such publications. The Applicant and the Institution shall notify without delay AFM’s Scientific Department whenever any article is published with the name of AFM in its acknowledgements and send a pdf to [email protected]. The Institution and the Applicant expressly authorize AFM to use, in the framework of communications relating to the Program, the name of [ORGANISME] and [DEMANDEUR] the amount of financing allocated by AFM to the Program, the title of the Program, to communicate the five-line non-confidential summary of the Program (lay summary) written by the Applicant in the application in English, as well as to describe the Program orally and/or in writing to the general public, notably during its campaigns for collection of donations, during the Téléthon and at the General Assembly of AFM, omitting details which might be prejudicial to the intellectual property rights of the results of the Program.

6. Progress Report(s)The Progress Report is defined as follows: It includes a scientific report and a financial report and shall follow the model presented as Annex 1 of the present document. The scientific report shall describe the scientific activities carried out in relation to the objectives of the Program, the ensemble of the results obtained during the previous year as well as the perspectives for the following year (if there is a request for renewal of funding). The financial report shall include (i) an assessment regarding the utilization of AFM funding for the previous year (if need be, any possible remainder of funding which has not been utilized during the period under consideration shall be refunded to AFM, or carried forward to the following year following a decision of the Board of Directors of AFM) and (ii) a provisional budget for the following year (if renewal for a second year has been requested).

For programs in which there is a request for renewal :In case AFM has accepted the principle of the financing of the Program for two (2) years, the Institution and the Applicant agree to integrate the Progress Report (incorporating an intermediate financial report) for the first year of the Program into the request for renewal during a Grant Application session, within a maximum time limit of eighteen (18) months from the date of signature of this Letter of Agreement (for more information on the procedure, the Applicant may contact [email protected]). Furthermore, within maximum a time limit of eighteen (18) months from the date of signature of the Letter of Agreement relative to the second year of the Program, the Institution and the Applicant agree to submit to AFM a final Progress Report as defined above (incorporating a final financial report covering the total duration of the Program).

For programs in which there is no request for renewal:If AFM has only agreed to finance one (1) year of the Program, the Institution and the Application agree to submit a Progress Report to the Permanent Secretariat of the Scientific Council of AFM within eighteen (18) months maximum from the date of signature of this Letter of Agreement. 7. TermThe present Letter of Agreement will take effect for a term of eighteen (18) months from the date of its signature. Expenses may be incurred by the Institution for the duration of the present Letter of Agreement, that is, for a duration of eighteen (18) months from the date of signature.

Articles 3, 4, 5 and 6 will continue in force after the expiration of the present Letter of Agreement.

Annex 1: “Model for Progress Report” is an integral part of the present Letter of Agreement.

Executed in Paris, on_____________

APPLICANTName (Last name, First name): Signature should be preceded by “Read and approved”

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SPCS / Permanent Secretariat of the Scientific Council96, rue Didot - 75014 PARIS France

Tel : 33 1 40 52 07 66 - Fax : 33 1 40 52 05 75

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Laboratory DirectorName (Last name, first name):Signature preceded by “Read and approved”

The Legal Representative of the InstitutionName (Last name, first name):Title:Signature preceded by “I declare to be duly authorized to represent the Institution, Read and approved”

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SPCS / Permanent Secretariat of the Scientific Council96, rue Didot - 75014 PARIS France

Tel : 33 1 40 52 07 66 - Fax : 33 1 40 52 05 75

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Annex 1Model of Progress Report

Scientific progress reporta) Describe the main goal and specific aims as described in the original application

b) Progress report: Describe in detail the accomplishments including all results, challenges, setbacks and explain, if applicable, the rationale for new directions taken. If the Project is a clinical trial, provide details of the progress of the trial (regulatory, patients enrolment, serious adverse events, …). At the end of the trial, send a copy of the final clinical report written for regulatory authorities.

c) Quantify progress made towards the initial aims

d) Next steps

e) Publications and /or Patents

List the publications acknowledging the financial contribution of AFM including papers submitted for publication, accepted for publication or in press (References must in-clude names of all authors, full title of the manuscript, name of the journal the manu-script has been submitted to and date of submission). Please send a copy of the ac-cepted papers to [email protected].

Intellectual property and patent(s) applicationThe applicant shall promptly notify AFM in writing of each Result and any patent applications filed thereon resulting from the research Project. (“Result” means any product, process, result, computer software, database, idea, information, development, conceived or created by the applicant and his/her program team, arising out of the research Project funded by AFM, that is or may be patentable or otherwise protectable by intellectual property rights).

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SPCS / Permanent Secretariat of the Scientific Council96, rue Didot - 75014 PARIS France

Tel : 33 1 40 52 07 66 - Fax : 33 1 40 52 05 75

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- 15 -ASSOCIATION FRANCAISE CONTRE LES MYOPATHIES

SPCS / Permanent Secretariat of the Scientific Council96, rue Didot - 75014 PARIS France

Tel : 33 1 40 52 07 66 - Fax : 33 1 40 52 05 75