Issue 22 - May 2019

Fabry Focus e-NewsletterMay 17, 2019

Greetings!

We hope our newsletter finds you doing well. We have a lot information to share with you since our last e-newsletter.

Now that we are well into spring in the northern hemisphere, we reflect on the many successes in the Fabry community in 2018 and thebeginning of this year and we look forward to so much more progress this year. It seems as though we are gaining clarity on so many importantissues ... even though new questions arise everyday.

April 2019 was Fabry Disease Awareness Month. We continually try to spread awareness but during the month of April we make an extraeffort. We posted an educational awareness post on our face book page at www.facebook.com/FabryDisease every day of April. The "reach"of each daily post (number of people who viewed each post) ranged from about 1,300 people to 11,000 people with an average post reach ofover 3,300 people. Our face book page followers are now more than 3,200 people overall. If you have not yet "Liked" and "Followed" our page,please do. Thank you for your tremendous support in spreading Fabry disease awareness!

This month's newsletter includes:

IntroductionApril 2019 Fabry Disease Awareness MonthOur Annual Fundraising CampaignApproval of Galafold in the U.S. 15th annual LSD World SymposiumNFDF Conference and Camp - Common application guidance for conference attendees, camp attendees, and volunteers 10th Annual Fabry Family Camp at Victory Junction - Phase 1 Application Process: Please complete Phase 1 now9th Annual Fabry Family Education Conference - Phase 1 Application Process: Please complete Phase 1 now2019 Camp Volunteer Application Information - Please complete Phase 1 nowPatient Reported Outcomes Survey Program

Winners from last surveyMay 2019 survey - adults with Fabry please participate for the benefit of everyone with Fabry disease!

University of North Carolina PodcastsWhat's going on in industry?Avrobio awareness month eventFamily Assistance Program via KGA, Inc. for counseling and referralsFinancial assistance programs for the Fabry communityCheck out the Amicus FabryConnect resourceNFDF resources - Educational symptoms calendars and My Health Handbook KitsNFDF Symptoms Presentation - PDF formatPatient Resource Education Packets (PREP)Shining Star Campaign donor acknowledgementsUntil Next Time

We are supporting individuals with Fabry disease and their families in as many ways as we are able. We are improving and adding to ourprograms and services as opportunities arise.

As always, we ask you to get involved and join the fight by participating in our many ongoing initiatives especially our Patient ReportedOutcomes Surveys (PROS). Your participation helps everyone with Fabry disease and our families!

Sincerely,

Jerry Walter

Fabry Disease Awareness Month

April was Fabry Disease Awareness Month!Congratulations and thank you to our state Fabry community representatives who submitted requests to obtainGovernor's proclamations! See who they are at this link:Community Representatives

We received governor's proclamations this year from the 35 states highlighted in green on the map below.

The states highlighted in yellow on the map are states we thought we would obtain this year but something went

The states highlighted in yellow on the map are states we thought we would obtain this year but something wentwrong. In some states the governors changed and their processes subsequently changed.

The states highlighted in red have requirements we cannot meet that prevent us from obtaining a proclamation.

To continue to spread awareness in your local areas, please print the flyer from the link below and distribute it wherever possible in places suchas your workplace or school; your local nephrology, cardiology, and neurology clinics; and local businesses. Also share the flyer on socialmedia. If possible, please let us know how many you print and distribute. Tag the NFDF in social media posts.

Fabry Disease Awareness Flyer - Get Tested

You can see photos of the proclamations and community members holding them in the "2019 Fabry Disease Awareness Month Photo Album"at www.facebook.com/FabryDisease.

Our Annual Fundraising Campaign - The fuel for our fire!

Our Annual Fundraising Campaign

You don't need a reason to help people but we have many of them if you arecurious about ours.

*We are giving adults and children with Fabrydisease more life to their years and more yearsto their lives!We have a tremendous opportunity to improve and even save the lives of

people with Fabry disease. We know many of you have made your 2019 annual contribution already. If you have not,please make a contribution during this annual campaign. Together we can make a huge difference!

*Note: Slogan above borrowed and adapted from the Belgium Fabry patient support organization.

Almost all the stars are aligned! We just need your help!

- There are many symptoms that can lead to Fabry diagnoses with increased education.- There is a definitive lab test to confirm a Fabry disease diagnosis.- There are treatments available now.- There are several more potentially improved treatments under investigation.

PLEASE JOIN THE FIGHT!

OUR ANNUAL FUNDRAISING CAMPAIGN IS MAY AND JUNE 2019.

PLEASE MAKE A CHARITABLE DONATION TO THE NATIONAL FABRY DISEASE FOUNDATION!

PLEASE MAKE A CHARITABLE DONATION TO THE NATIONAL FABRY DISEASE FOUNDATION!

Contributions can be made by mail to:

NFDF NC OfficeP.O. Pox 1325

Carrboro, NC 27510

Contributions can be made online through our Network for Good donation page at the Donation button below:

Or donations can be made on our face book page at www.facebook.com/FabryDisease. There are no fees on face book.

Hold a face book fundraiser, another type of fundraiser, or make an automatic recurring donation for a greater impact!

See the bottom of the newsletter for a list of our 800+ donors since our last e-newsletter was distributed.

Thank You!U.S. Approval of GalafoldTM

On August 10, 2018, Galafold was approved as the second treatment forFabry disease in the U.S.

For 15 years there was only one approved treatment for Fabry disease in the U.S., the Enzyme ReplacementTherapy Fabrazyme. We are very happy to report that In August 2018 a second treatment, the oral chaperone therapy Galafold, wasapproved. In many other countries Fabrazyme, Replagal and Galafold are all approved.

We are very fortunate to have had enzyme replacement therapy for so many years but our community is not a one-size-fits-all community.

Adults with Fabry can work with their physicians to determine which treatment is more appropriate based on each individual's circumstancesand needs. People with amenable mutations are candidates for treatment with Galafold. To find out if your family mutation is amenable askyour Fabry health care specialist or contact Amicus at the link below.

For patients: http://www.amicusassist.com/home/for-patients/

For healthcare providers: http://www.amicusassist.com/home/for-healthcare-providers/

If you have trouble finding out, let us know. We may be able to help.

The Annual Lysosomal Storage Disease World Symposium

15th Annual WORLD Symposium

On February 3rd to February 7th, Susan Spinks, who works with me at the National Fabry DiseaseFoundation, and I attended the 15th annual World Lysosomal Storage Disorder Symposium in Orlando,FL. There were hundreds of attendees from medical institutions, the pharmaceutical industry, and non-profit patient support organizations.

We had a busy week listening to research updates about Fabry disease in the educational sessions;meeting with pharmaceutical company representatives about current and potential treatments for Fabry

disease and about their patient/family programs; meeting with other patient/family support group representatives; and distributing our PatientResources Education Packets to Fabry providers (physicians, physician assistants, and nurse practitioners) and clinic staff (geneticcounselors and nurses).

While there, I also attended the Annual Council of Patients Advocates meeting.

What we learned and accomplished at the World meeting helps us to keep you informed and will help to guide our education, awareness, andsupport programs throughout the year.

Jerry Walter

For Prospective Camp Attendees, Conference Attendees, and Camp Volunteers

Let the Games Begin!

Phase 1 of the Conference and Camp Application Process

The starting point (application process phase 1) is the same whether you are a family applying to attend both our September camp andconference, a family or individual applying to attend the conference only, or you are an individual submitting an application to support camp asone of our awesome volunteers.

As you read the separate guidance sections below, everyone please complete Phase 1 first by submitting your family or individualinformation to Jerry at jerry.walter@fabrydisease.org as soon as possible.

Here is what we need. Please ensure you send all the information requested in one email if possible to avoid having multiple messages totrack.

- Please tell us which of the following events do you intend to submit an application for?1. to attend the conference and camp (individuals with Fabry disease and family members)2. to attend just the conference only (individuals with Fabry disease and family members not attending camp also)3. to volunteer to support camp

Please provide the following information:

- the first and last names of all camp and/or conference attendees in your family and if you are a prospective volunteer (For adults, provide names as they appear on the ID you'll use at the airport (submit names even if you are driving)).- Preferred names for name tags- Birth dates- Gender- School grade for school age children with families- Who in your family has Fabry?- Dietary restrictions, if any, for everyone - Preferred airport you wish to fly from or indicate that you are driving(unless you are from industry and your company provides your travel)- Cell phone you will use while traveling- Home address- Prospective volunteers from clinics and industry - provide your clinic or company name.- Individuals with Fabry - Does your family have a classic or non-classic disease mutation? Please provide your family mutation. See pages 15-18 of ourFabry Community Landscape Handout at https://www.fabrydisease.org/.../The-Fabry-Disease-Community-.... If you are unsure about what it is, ask yourFabry physician. - Any special needs

See additional information in the separate guidance sections below.

Attention industry representatives, health care providers and clinic staff, infusion providers:

Please ensure individuals with Fabry disease you are in contact with know about our Septemberconference and camp. Please share my contact information: Jerry Walter at 800-651-9131 or jerry.walter@fabrydisease.org. Phase 1 of the application process has started.

Start Your Engines - Feel Your Heart Race

10th Annual Fabry Family Camp Guidance

Our camp at Victory Junction is is 6-8 September, 2019.

Families - Complete phase 1 of the application process (above) as soon as

Families - Complete phase 1 of the application process (above) as soon aspossible and before you complete phase 2 when the online application opens

sometime in June to be determined (TBD).

Our 10th annual expense-free Fabry family camp at Victory Junction will be held from Friday, September 6, 2018 (5:00 pm) until Sunday,September 8, 2019 (noon) right after our pre-camp conference on September 5 -6, 2018 in Greensboro, NC. The camp in Randleman, NC isabout a 40 minute drive from the conference hotel in Greensboro, NC.

In addition to proving airfare, lodging and most meals, we provide transportation from the conference hotel to camp and back to the airport onSunday. Our conference hotel has a free shuttle from the airport to the hotel. Camp attendees are automatically signed up for the precedingThursday - Friday conference. Some families will travel to the conference on Wednesday rather than Thursday morning depending on availableflight times.

To attend camp, families must have at least one child with Fabry disease between ages 6 and 16 (called the Primary Campers) forthe entire immediate family to attend camp. We can accept 32 families each year. All individuals/families in our community are invited toattend the pre-camp conference, not just families with eligible children going to camp. See the conference guidance below.

Online camp applications will be accepted from the start date we will announce soon (sometime in June) until July 31st. However,please apply as soon as possible after the start date is announced. Do not wait until the final application due date so we avoidcomplications. The camp application approval process takes some time. Once approved, we'll purchase plane tickets.

Phase 1 - The first step (above)

Phase 2 - Victory Junction's online application - Start date to be announced

- Victory Junction is changing their process. Soon, when we have the revised 2019 application guidance from Victory Junction, we will send itto everyone who has completed Phase 1 by providing your family information. We look forward to seeing you soon!

9th Annual Fabry Family Education Conference

Join us at our 2019 Fabry Family Education Conference Our 9th annual Fabry Family Education Conference will be held on Thursday, September 5, 2019 beginning atnoon until Friday, September 6, 2019 ending about 4:30 pm just before families depart for camp.

Individuals with Fabry and family members DO NOT have to attend camp to attend theconference. Hotel lodging and most meals are provided. Travel assistance may be provided by the NFDF

upon request. When families going to camp depart Friday afternoon, individuals and families not going to camp will depart for home. Someattendees may have Saturday departures instead of Friday evening departures depending on available flights.

Conference attendees will begin arriving at the conference hotel around noon on Thursday and will continue arriving throughout the afternoonand evening depending on flight schedules. Some families may travel on Wednesday if you have difficult itineraries.

Our conference hotel this year is the Greensboro Airport Embassy Suites Hotel at 204 Centreport Dr, Greensboro, NC 27409 Phone: (336)668-4535. We make your hotel reservations and provide family hotel rooms. Please do not make your own reservations.

We'll provide a conference agenda a little later once all physician speakers and other speakers have been confirmed. The conference application process is not as formal as the camp application process but the starting point (phase 1) is the same.

Conference only attendees - Please provide the phase 1 information above as soon as possible. We look forward to seeing you at the conference!

Camp Volunteer Applications

Camp Volunteer Applications - Thank you for your support!

The camp volunteer application process begins with phase 1 above.

The camp volunteer application process begins with phase 1 above.

We will know the official start date for phase 2 (the online application) soon (afterMay 16th) and will let you know when we have it (start date will be sometime in

June TBD).

Each year many family members, friends, clinic staff, industry supporters, and others help to make our camp possible. We help facilitate theVictory Junction application process to obtain about 60 Fabry community volunteers.

We would love to have you join us as a volunteer if you are healthy enough to perform all the requirements. We walk a lot around camp (it'ssomewhat hilly), work fairly hard, and have a couple of long days but we have a great time together supporting our families. Volunteerapplicants must be at least 19 years old. Most volunteers are family crew chiefs with a few exceptions as you'll see in the online application.Please let us know if you are willing to help with food services in the dining facility/fuel stop as your primary job with assistanceto families during non-meal times.

The volunteer application process is managed by Victory Junction (VJ). VJ has simplified their process this year. Medical exams are nolonger required and the number of required immunizations have been reduced. The application process still has several steps sostarting your application early is critical once we announce the start date for online applications. Final application approval is determined jointlyby the VJ volunteer coordinator and the NFDF.

We will provide the online application start date in mid May as soon as Victory Junction provides it. It will likely be July 1st but possibly sooner.When the online application opens, please complete it as soon as possible. Please make medical appointments for requiredimmunizations and/or titer tests as soon as possible.

The first step in the process is to notify Jerry from the NFDF at jerry.walter@fabrydisease.org that you intend to submit anonline volunteer application to Victory Junction. Your application will be delayed if the Victory Junction volunteer application listdoes not coincide with NFDF's volunteer list. Please provide the phase 1 information above as soon as possible and before the online application (phase 2). There are new medical requirements this year:

- 2019 VJ medical requirements - Annual physicals are no longer required.

If you are an industry volunteer, your company may have additional requirements. Please inform/coordinate with your appropriate manager andvolunteer coordinator as appropriate but not in lieu of adhering to NFDF requirements. Please copy your company volunteer coordinator whenyou send your phase information.

We really appreciate your tremendous support!

Thank you! Jerry Walter

Patient Reported Outcomes Survey (PROS) Program

Patient Reported Outcomes Survey (PROS) program - Learning from the PROS

If you have Fabry disease, please help us to gather information to continue learning about Fabry disease and tosupport development and implementation of important education and community support programs.

The results of past surveys have provided great benefit to people with Fabry disease by providingimportant information and insights.

We implement the PROS program in a contest format to encourage participation and to make it more interesting for participants.

You must be 18 years old or older by the end of 2019 with the Fabry gene (with or without symptoms) to participate in the survey program. Weencourage worldwide participation but regret we are only able to provide the survey program in English at this time.

We do not share personal information except for first names and broad locations of prize drawing winners.

The results of the June 2018 survey are at this link: June 2018 survey results

__________________________________________________________________________________________________________

The winners of the June 2018 survey (the last survey we distributed) are:

U.S. winners ($250.00 USD) - Angela from Raleigh, NC and Veronica from Chula Vista, CA

Non-U.S. winner (€223 Euro) - Charlotte from Cork, Ireland

Winner of 2018 End of Year drawing ($500.00) - Sue from Somers Point, NJ

__________________________________________________________________________________________________________

__________________________________________________________________________________________________________

To begin the May 2019 survey please select the link below. Share it with other adults with Fabry disease.

Please participate to continue to improve our understanding of Fabry disease and of the Fabry community.

May 2019 PROs Survey The results we collect and share with you will be much more representative of the overall Fabry community and much more useful if we have alarge number of participants. Your participation can help our entire community.

Please take the 2019 surveys by their due dates to be eligible for each survey's prize drawing!

For each survey distributed this year, a prize drawing of $250.00 will be awarded to two U.S. participants and one global (non-U.S.) participant.Please share this information with your adult family members with Fabry.

After the end of the initial survey period, each survey will remain open through the end of the year so you may still take it to be eligible forthe end-of-year $500 prize drawing for everyone who completes all the 2019 surveys by the end of the year. If you cannot accept the prize because of income limitations if you receive social security benefits or for other reasons, please still take thesurveys to improve the information collected. Just let us know you cannot accept the prize. Thank you for your support and good luck!

Other news

University of North Carolina podcasts about Fabry disease The NFDF is always looking for new ways to provide education and awareness about Fabry disease. Jerry

Walter participated in two University of North Carolina podcasts with Dr. Ron Falk (Director of the UNC

Kidney Center) Dr. Jerry Hladik (UNC Chief, Division of Nephrology and Hypertension).

Thank you to Dr. Falk and Dr. Hladik for this great opportunity to spread awareness about Fabry disease.

https://www.med.unc.edu/medicine/news/chairs-corner/podcast/fabry-disease/?fbclid=IwAR2ZeGIKAuJluQ29hvj0R2vX-qg0Rtb9-WlDIl_wyOxMgYxBzt-11K1-wxk

https://www.med.unc.edu/medicine/news/chairs-corner/podcast/galafold/

Call us today to reach yourCareConnectPSS Case Manager.

1-800-745-4447, Option 3

The Pharmaceutical IndustryWhat's going on in industry?

Are you interested in participating in a clinical trial?A tremendous amount of research has been done and continues to be done about Fabry disease. As a result, we arevery fortunate to have three treatments approved for Fabry disease around the world and two treatments available inthe U.S. Many people with Fabry disease are living longer. Many people with Fabry disease are being treated earlierin life and avoiding some of the many complications of Fabry disease. Our future looks brighter than in the past!

As a community we are better off today than in the past because people selflessly participated in clinical trials. Yet,there are still so many questions to be answered and many problems yet to be solved. We believe it can still get muchbetter.

If you have Fabry disease and are interested in participating in a clinical trial to continue making progress towardbetter solutions and better and longer lives, let us know at info@fabrydisease.org. We'll help to connect you to the various physicianinvestigators conducting the trials you are interested in. We can't help you decide which clinical trial may be right for you but we can help youonce you work with your physician to decide if a trial may be right for you.

Together, we can make a difference! Jerry Walter

Personalized patient support services for you!

INTRODUCING CareConnectPSS

Personalized support for patients and families affected by rare disease

CareConnect Website

CareConnectPSS is designed to support each patient's unique journey to help you overcome challenges related to living with a rare disease.

The CareConnectPSS team includes Case Managers and Patient Education Liaisons who are dedicated to providing one-on-one assistance.

ASPIRE Pediatric study for Migalastat approval

Amicus Therapeutics is enrolling children and teens in their ASPIRE pediatric study for potential approvalof the oral medication migalastat for children.

If there is not a study site nearby, you may be eligible for travel assistance to participate.The trial sites are located at: - University of South Florida in Tampa, FL- Emory University Div of Medical Genetics in Atlanta, GA- University of Missouri in Columbia, MO

- University of Missouri in Columbia, MO- Cincinnati Children's Hospital in Cincinnati, OH- Children's Hospital of Philadelphia in Philadelphia, PA- Children's Hospital of Pittsburgh of UPMC in PA- Lysosomal & Rare Disorders Research & Treatment Center in Fairfax, VAFor more information contact: Amicus Therapeutics Patient Advocacy at 609-662-2000 or clinicaltrials@amicusrx.com.

Link: ASPIRE Study Brochure

News about Replagal Enzyme Replacement Therapy

To help to keep our non-U.S. friends up to date, we are sharing the news that the acquisition of Shire(manufacturers of Replagal) by Takeda Pharmaceutical Company has been completed. We look forward to

continuing our relationship with Takeda Shire as we support the global Fabry community.

Replagal is available in many countries around the world but is not available in the U.S.

The Protalix Balance Study is continuing to enroll participants.

Protalix continues to enroll participants in their Balance and Bright (non-U.S.) clinical trials for the investigational Fabry disease treatment PRX-102.

Here is an excerpt from a press release recently issued by Protalix: "Throughout 2018 and into early 2019, wesignificantly advanced our clinical development program for PRX-102 and, as of today, have enrolled 127 Fabry

disease patients across all of our PRX-102 clinical trials. Most recently, we had a very productive meeting with the U.S. Food and DrugAdministration (FDA) to discuss the potential filing of an application for accelerated approval and, at the FDA's request, we intend to hold afollow up meeting by the end of the second quarter of 2019 to discuss the data and content of the potential filing for accelerated approval.... "

See details at: http://www.fabrynext.com/

Link: Protalix Balance Study Brochure

See this link ClinicalTrials.gov (for criteria and study locations)

Lentiviral Based Gene Therapy Trial for Fabry Disease

AVROBIO, a leader in lentiviral-based gene therapies, is a clinical stage company developing disruptivetherapies that have the potential to transform patients' lives in a single dose. Gene therapy involves the transplantation of normal genes into cells in order to correct genetic disorders. Gene therapy uses a vector (seeillustration below) to carry and deliver a working copy of a gene to the body's cells. A vector is a deactivated virus that is unable to reproduceand spread. There are different types of vectors. AVROBIO's plato™ is a gene therapy platform which uses a lentiviral vector and is designedto target underlying genetic disease by restoring normal gene function and enzyme production.For more information about AVROBIO's pipeline and technology please visit https://www.avrobio.com/.

FDA Clearance of Investigational New Drug Application

AVROBIO, Inc. Announces FDA Clearance of Investigational New Drug Application for AVR-RD-01 Gene Therapy for the

Treatment of Fabry Disease The plato™ platform represents a significant advance towards a commercial-stage gene therapy solution designed to treat thousands ofpatients . AVROBIO to incorporate U.S. clinical sites into its ongoing global FAB-201 Phase 2 clinical trial in Fabry disease

Here's a link to the full BusinessWire news release:

https://www.businesswire.com/news/home/20190429005136/en/AVROBIO-Announces-FDA-Clearance-Investigational-New-Drug

ClinicalTrial.gov - Australian trial underway

FDA Acceptance of IND Application for ST-920 Gene TherapyCandidate for Fabry Disease.

Sangamo Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted the Investigational New Drug (IND)application for ST-920, a gene therapy candidate being evaluated for the treatment of adults with Fabry disease. Current standard of care forthis rare, progressive condition involves regular lifelong infusions of enzyme replacement therapy (ERT).

The company plans to initiate the Phase 1/2 clinical trial for ST-920 in 2019.

https://www.sangamo.com/

How Sangamo gene therapy works

Lucerastat, a new oral therapy in development for Fabry patients

Idorsia initiated MODIFY, a Phase 3 registration study to assess lucerastat as a potential new treatment option forpatients with Fabry disease.

Lucerastat is an oral Substrate Reduction Therapy (SRT).

Enrollment is now open. See the clinical trials link below to view criteria and study locations. Travel assistance and reimbursement areavailable.

News Release Link

ClinicalTrials.gov link (for criteria and study locations)

Other News and Resources

Fabry Disease Awareness Month Event

We were honored to be invited by Avrobio to visit them in Cambridge, MA during Fabry DiseaseAwareness Month.

On April 25th Jerry Walter, NFDF, and Kim McCarthy, a MA Fabry community representative, visitedAvrobio to learn from each other and to celebrate Fabry Disease Awareness Month. Kim brought theMA Fabry Disease Awareness Month proclamation signed by Governor Charlie Baker that sheobtained on behalf of Fabry community members in MA.

About 35 Avrobio staff members and leadership were present as well as other staff members on aconcurrent audio call. We had a great visit!

We look forward to hearing more from Avrobio as they advance their gene therapy clinical trial for Fabry disease.

Thank you to President and CEO Geoff MacKay and his team for spending some quality time with us and thank you to Fernanda Copeland,Chief, Patient Advocacy, for organizing the event.

We had a great visit!

Free Family Assistance Program

The NFDF is proud to continue our free, confidential, 24/7 Family Assistance Program for individuals in the U.S.with Fabry disease and their family members.

The NFDF contracts the confidential professional counseling services of Kathleen Greer Associates (KGA) who are standing by ready to takeyour calls. Any personal issue is appropriate. The service is completely confidential. The NFDF only receives summary data about theassistance provided by KGA, no personal information.

To reach a counselor 24/7, call 800-648-9557. See more details in the Featured Programs section on the NFDF website homepage at www.fabrydisease.org.

Financial assistance for people with Fabry diseaseThere are three primary financial assistance programs available to people with Fabry disease.

Patient Services Incorporated (PSI) at https://www.patientservicesinc.org/

The Assistance Fund (TAF) at https://tafcares.org/

The PAN Foundation at https://panfoundation.org/index.php/en/

Both the TAF and PAN Foundation open and close periodically throughout the year depending on availableresources.

For TAF, even though the fund opens and closes periodically, once a person is approved in a calendar year their assistance is approved forthe whole year.

In addition, Sanofi Genzyme has a co-pay program and both the NFDF and FSIG have small short-term financial assistance programs whenassistance is not available from the three primary programs.

Check Out FabryConnect

Amicus Therapeutics has recently launched FabryConnect, a website intended as a resource for people living with Fabry disease and theirfamilies.

Visit: http://www.fabryfacts.com/ and https://fabryconnect.fabryfacts.com/

- Listen to webinars on relevant Fabry topics of interest. - View videos and stories of others living with Fabry disease. - Download Fabry brochures and educational materials for yourself or to share with family/friends, physicians and other healthcareprofessionals.

This is a dynamic site intended to provide new information and educational content on a regular basis with input from the community.Additionally, Amicus is open to advice and suggestions on how to improve appearance, usability, interactivity and value. Navigatethrough https://fabryconnect.fabryfacts.com and provide feedback on what you would like to see or read about.

If you have Fabry disease, do you have your calendar and kit?

If you have Fabry disease, do you have your calendar and kit?

Do you have an Educational Fabry Symptoms Calendar and a My Health Handbook Kit?

For the U.S and international Fabry community

Our 2018-2019 calendars are still available.

The My Health Handbook Kit includes:

a durable, stylish carrying case our My Health Handbook a 8 GB credit card style USB drive alert cardwristbands and other awareness materials

To obtain a kit and a calendar, follow the guidance in this flyer . If you havequestions, email jerry.walter@fabrydisease.org.

If you work at a Fabry clinic, please request copies of our PatientResource Education Packets (PREP). Ensure you patients are PREPed for theirjourneys with Fabry disease. We also provide a sample kit and calendar to clinicproviders and staff who see people in clinic to show as examples of our availableresources.

Our PREP includes the flyer to the left as well as:

A copy of our Programs Handout also located at https://www.fabrydisease.org/images/National-Fabry-Disease-Foundation-Programs-Handout-Nov-18.pdf

A copy of our Fabry Community Landscape Handout also located athttps://www.fabrydisease.org/images/The-Fabry-Disease-Community-Landscape-Handout-Nov-18.pdf

Educational Fabry Symptoms Presentation

We converted our educational symptoms calendar images into a PDF for easier access. This is a great tool to better understand themany common symptoms of Fabry disease and to educate family, friends and the public.

The PDF file is located on our website at https://www.fabrydisease.org/FabryDiseaseSymptomsBriefing.pdf

Patient Resource Education Packets (PREP)

Providers and clinic staff: Are all individuals with Fabry and their family members PREPed fortheir journeys with Fabry disease?

One of our primary goals for this year is to strengthen our relationships with Fabry diseaseproviders and their staff. This effort will help us in at least three ways. 1) We want to ensurethat every family with Fabry disease receives information about NFDF programs andservices to give families an opportunity to benefit from them. 2) We would like to collaboratewith clinics to ensure we are meeting the needs of families from their perspectives. 3) Wewould like to add all primary Fabry providers who manage and treat Fabry disease to ourwebsite Find A Specialist database.

We have already sent hundreds or packets to clinics and to individuals. We have hundredsyet to send.

If you are a provider or staff member at a clinic managingsomeone with Fabry disease and you have not already receivedsample packets (and sample My Health Handbooks and calendars) toshow your patients so your they can request their own, pleasecontact us at jerry.walter@fabrydisease.org.

An Essential Ingredient to our Success is You!

Shining Star CampaignBe the brightest star you can be!

Thank you to our generous shining stars for this period! This list contains donors who made charitable contributions to

the NFDF since our since 2018 e-newsletter was published.

Our Sirius Sustainers (Major Contributors) (Read about our *Sirius Sustainer Program below)

(Read about our *Sirius Sustainer Program below)

Sanofi GenzymeAmicus Therapeutics

Protalix BiotherapeuticsAvrobio

Sangamo TheraputicsIdorsia Pharmaceuticals

Champion Level Donors ($1,000 or $9,999)

Fundraiser by Shirley Lewis of California Groomin'Diplomat Specialty Infusion GroupData Registry ServicesJeff MinelliSteve and Diane RothmanStacy Ho plus a matching company contributionFred (Freddie) Sico Jr. and his wife Michelle in memory of Michael Sico (see correction below)

Correction from our last newsletter: We incorrectly listed a Champion Level donation by Lynn Boudreau. Lynn provided the information for the donation made by Fred Sico Jr. andhis wife Michelle. We apologize for the error.

Sustainers: (Automatic Recurring Donations)

Jaime Hanrahan in honor of the Bohn FamilyErin Devine with a company match from Cambria Health Foundation Trinh Kelleher with a company match from Ameriprise FinancialAnonymous via United Way of Central New MexicoSarah Lopez via PIMA County ECAP

Leader Level Donors ($500 to $999)

Facebook Fundraiser by Jason BackFacebook Fundraiser by Kellie JohnsonFacebook Fundraiser by Kelly StadelmanFacebook Fundraiser by Phil HeidrickJohn KingBob and Beth Orscheln in memory of Matt JonesJay Marshall in honor of Beverly BrinleyJean and Steve Yabroff in memory of Beverly BrinleyJean and Steve Yabroff in honor of Blake JohnsonGroup donation: Jr. and Judy Brinley, Bill and Eva Brinley, Beverly Routh, Traci Newton, Brian Newton and Jason Newton, in memory of BeverlyBrinley

Complete Donor List Continued (select the link above)

Since our last e-newsletter in June 2018, we received nearly 800 individual donations. Many of thosedonations were a result of about 120 face book fundraisers in addition to our mail-in and other onlinedonations. Please select the link above to see the complete list of our donors since our last e-newsletterpublication.

*Sirius is the brightest star (in fact, star system) in the Earth's night sky. Our Sirius category donors makemajor contributions to provide our annual funding base on which to build. Our Sirius sustainers providethe starting point to make our programs possible.

Thank you to all of our wonderful donors for your amazing support! Every size contribution adds up tomake a huge difference. You have our deepest gratitude for being the brightest stars you can be!Together were better!

Fabry Disease - A road less traveledUntil Next Time

Thank you all for your continued participation and support! Please contact me anytime at the phone number or emailaddress below.

address below.

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Proudly serving the Fabry community!

Sincerely, Jerry Jerry Walter, MSColonel, U.S. Army (Retired)Founder and PresidentNational Fabry Disease Foundation

www.fabrydisease.org

Copyright © 2019 All Rights Reserved.

| 800-651-9131 | jerry.walter@fabrydisease.org | 4301 Connecticut Ave. N.W.

Suite 404Washington, DC 20008