Dear friends,
We are proud to share that for the 11th consecutive year, FARA has been recognized by Charity Navigator as a 4-star charity, their highest rating! This places FARA among the top 2% of charities evaluated by Charity Navigator, who base their ratings on a nonprofit's financial health and commitment to accountability and transparency. Click here to read more about FARA's rating. Thank you to the entire FA community for your generosity of time and resources which strengthen the organization and research effort.
Sincerely,
Jen
Jen Farmer,
Chief Executive Officer
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FARA Newly Funded Research |
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Three-dimensional mature cardiac microtissues from human induced pluripotent stem cells to explore mitochondrial dynamics, cardiac function and therapeutic options in Friedreich Ataxia
Paola Costantini, PhD - University of Padova, Italy
In this project the investigators will use an innovative tool, cardiac microtissues or “mini-hearts”, obtained from patient stem cells to investigate 1) if remodeling of the mitochondria can correct cardiac dysfunction and 2) if increasing the levels of frataxin can correct the mitochondrial and cardiac defects. Modeling cardiac dysfunctions in Friedreich ataxia is a crucial and challenging task and cardiac microtissue is an advanced cellular model that will allow us to mimic the human heart in a dish.
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Novartis Gene Therapies Announcement - Novartis Gene Therapies announced that they have received Orphan Drug Designation from the FDA for OAV401, and that they expect to submit an Investigational New Drug (IND) application by the end of this year. An active IND will allow them to initiate the clinical investigation of OAV401 for the treatment of FA.
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The Minutes of Science series continues with Episode 3: Drug Targets in the Central Nervous System
Continue to tune into the series with new episodes debuting every month! You can also watch the entire series on the FARA YouTube page here. |
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2021 rideATAXIA Coastal Challenge |
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REGISTRATION IS NOW OPEN!!
Come join the Gainesville and SoCal communities in our virtual cruise happening soon!
The two coast communities will be going head to head in fundraising, community activity mileage, and social media engagement to see who will be declared the winner of our 10-day virtual cruise campaign. We set sail on March 17th, so register TODAY!
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FARA Energy Ball -September 25, 2021 |
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Save the Date for The FARA Energy Ball on September 25, 2021. Whether you join us for the event in Tampa, FL or stream it remotely, you can join in the fun! Stay tuned for more details.
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Mark your calendar for these 2021 Grassroots Events:
Well Crafted Kitchen Pizza Food Truck Fundraiser
April 11, Rodgers Forge Tot Lot (6531 Blenheim Road, Baltimore, MD)
$1 from every pizza sold will go to FARA
Cure FA Soirée
May 22, Edmond, OK
Flatbread Pizza Night
June 1, Bedford, MA
Claxton Classic Tournament
August 13, Maryland
Fuzzy Buzzy Golf Tournament
September 12, Windham, NH
Gasparilla Concours d'Elegance
December 3-5, Lutz, FL
**In support of GCDE's philanthropic efforts, a generous supporter has posed a $25,000 matching gift challenge! To make a gift to GCDE that will be matched dollar for dollar, please visit http://bit.ly/3aSTabd**
More is in the works for 2021! Please contact ann.musheno@curefa.org to start planning your grassroots event or to put a Save the Date on our events calendar!
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New Advocacy Newsletter & Website |
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FARA just launched a newsletter dedicated to Advocacy News to keep you up to date on the latest initiatives as well as education and outreach opportunities. Click here to read the first issue.
Also, be sure to check out the new Advocacy Website containing comprehensive information on this year's initiatives and how to take action. |
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Exercise Study at CHOP and University of Pennsylvania
Currently, 15 participants needed for this study.
Researchers at the Children’s Hospital of Philadelphia and the Hospital of the University of Pennsylvania are recruiting participants for a study to test the effects of a dietary supplement combined with exercise on aerobic capacity. Aerobic capacity refers to the body’s ability to take in, transport, and use oxygen during exercise.
Eligibility includes:
- Documented genetic diagnosis of FA
- Age 10 to 40
- Ability to complete exercise testing
- Not pregnant for women of child-bearing years
If you are selected to participate in this study, you would complete 2 in-person study visits, each lasting 2 days, over a 12-week study period at CHOP and Penn. During the study period, you may complete blood testing, exercise testing, administration of a dietary supplement, MRI scans, oral glucose tolerance testing (with a non-FDA approved stable isotope product) and optional muscle biopsies.
You will be compensated for your time.
To learn more about the study and determine your eligibility, contact the
(267) 425-1998.
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*NEW* FA-Health Index survey – Phase 3 with interview
Individuals with FA, age 11 and older, are invited to participate in this study.
The study involves completing an online survey and participating in a phone interview. The survey will take approximately 20 minutes to complete and will ask some personal questions about you, your household, and how FA affects your quality of life physically, mentally, and emotionally. After taking the survey, the interview will take approximately 60 minutes and will discuss how easy it was to complete the survey, how understandable the questions were, the format of the survey, and what some of the questions mean to you.
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*NEW SITES OPEN* PTC-743: MOVE-FA Study
Enrollment now open at Children’s Hospital of Philadelphia (CHOP), University of South Florida, UCLA, Murdoch Children’s Research Institute (Australia) and CHUM (Canada).
MOVE-FA is a Phase 2/3 interventional study with planned enrollment sites in Australia, New Zealand, Brazil, Canada, the U.S. and throughout the EU.
Eligibility criteria include:
- Genetically confirmed FA with two GAA repeat expansions (currently, point mutations and deletions are excluded from the study)
- Children ages 7-17 and Adults age 18 and older
- Ability to walk 10 feet in one minute with or without assistance
- Be able to swallow capsules
- Not have participated in another interventional study in the 2 months prior to enrollment in MOVE-FA
This interventional study will monitor the safety and efficacy of the drug Vatiquinone, which is expected to decrease the activity of an enzyme that allows free iron to trigger a type of programmed cell death that occurs more frequently in people affected with FA.
Information on MOVE-FA can be found HERE. |
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Heart and Eye Study at Weill Cornell Medical College
20 participants needed
This study is designed to characterize the cardiac manifestations of FA using exercise, MRI, ECHO and blood tests. Additionally, this study will include corneal confocal microscopy (CCM), a non-invasive eye evaluation.
Eligibility includes:
- Confirmed diagnosis of FA
- Age 12 – 17 plus an unaffected sibling in the same age range
Study participants and their siblings will have 2 visits in 1 year at Weill Cornell Medicine in New York City; rapid COVID-19 testing will be performed at the hospital prior to study visit. Financial support is available for travel and hotel. For eligibility screening and additional study information, please contact the study coordinator through email at cora@med.cornell.edu
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IDEA Study for Wearable Device
Participants can be involved in an investigational drug trial and still be eligible for the IDEA study. Participants are needed at all sites in Baltimore, Boston, Chicago, Los Angeles or Portland (OR).
The IDEA research study is testing body-worn sensors to measure movement. The goal of the study is to evaluate progression and severity of ataxia. The study involves visits every 6 months over 2 years at a clinic site. Participants must be 12-30 years of age and be able to walk 10-feet independently, without an assistive device. Involvement in the study provides compensation for travel and an Apple watch.
For additional eligibility criteria, please contact study coordinator Hannah Casey
( hannahcasey@uchicago.edu) or (773) 702-4610. Please specify your city of interest for enrollment - Baltimore, Boston, Chicago, Los Angeles or Portland (OR). |
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Anyone considering participating in a clinical trial should discuss the matter with his or her physician. FARA does not endorse or recommend any particular study. |
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The goals of the FA Global Patient Registry (FAGPR) are to collect information on all FA patients in one registry, to develop the registry into a powerful resource for research, and to engage the FA community in studies aimed at advancing our knowledge of FA and the treatments being developed.
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The Pathway
You can help fund research progress all year by joining our monthly giving program, The Pathway. When you join The Pathway with a monthly donation of $10 or more, you are joining a community invested in setting a path to treatments and a cure for FA through research. Click the image to give today.
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