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News & Press Releases

Here you will find the most recent organizational news from FARA, including information on events, and awareness and advocacy initiatives. To locate an article from a certain date, please use the archives on the right side of your screen.


Central Nervous System Therapeutic Targets in Friedreich Ataxia

Ian H. Harding, PhD, David R. Lynch, Arnulf H. Koeppen, and Massimo Pandolfo, MD
Published in Human Gene Therapy, 25 Nov 2020 https://www.liebertpub.com/doi/10.1089/hum.2020.264

Because many of the major targets of long-term therapy for FA are in the central nervous system (CNS), FARA worked with several FA investigators to write an in-depth review of the pathology present in the brain and spinal cords of patients with FA. The diverse group of experts was able to evaluate evidence gathered from human clinical observations, physiological, pathological and imaging approaches, as well as studies in animal models. The resulting paper provides detailed summary of the current understanding of what areas of the spinal cord and brain are impacted by the disease, including what specific cells are vulnerable to the loss of frataxin.

Moreover, the review also provides some insight into the temporal course of neuropathological changes in the CNS, which helps to provide information on areas that may respond to therapy and be successfully treated at different timepoints during the progression of the disease.

This comprehensive review of the impact of FA in the CNS is useful to patients, caregivers, physicians, as well as drug developers, especially those planning to test therapeutics aimed at the spinal cord and/or brain. FARA effort to organize and support production of this review represents the ongoing commitment to focusing resources on filling gaps in our understanding of FA and building relationships needed to find effective treatments.

December 1st is Giving Tuesday!

Giving Tuesday
December 1st marks #GivingTuesday, a global day of giving fueled by the power of social media and collaboration. On December 1st and throughout the holiday season, consider giving a gift to FARA as a #GiftofResearch to the entire FA Community!

Your gift of research will provide research tools and insights into Friedreich's ataxia. As 2020 draws to a close, the combination of increased research opportunities and fewer fundraising events presents us with a unique challenge. This Giving Tuesday, give the Gift of Research to help us close the funding gap and accelerate research momentum.

Please mark your calendar for December 1st, to either:

Give the Gift of Research
 
Create a Fundraiser

PTC Therapeutics Announces Initiation of Global Phase 3 Clinical Trial to Evaluate Vatiquinone in Friedreich Ataxia

- Third trial initiated in 2020 from compounds identified from PTC's Bio-e platform -

SOUTH PLAINFIELD, N.J., Nov. 30, 2020 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT), today announced the initiation of the registration-directed Phase 3 MOVE-FA study evaluating vatiquinone (PTC743) in children and young adults with Friedreich ataxia (FA). FA is a genetic, progressive, neurodegenerative movement disorder, typically diagnosed in childhood or adolescence.1 PTC estimates that there are 25,000 patients with FA worldwide and there are currently no approved disease modifying therapies for FA. In a previous Phase 2 trial, vatiquinone demonstrated a statistically significant effect on disease severity at 24 months relative to age and stage-matched natural history controls as assessed by the validated Friedreich ataxia rating scale (FARS) score and a favorable safety profile. Vatiquinone has been granted Orphan Drug Designation and Fast Track Designation for Friedreich ataxia by the U.S. Food and Drug Administration (FDA).

Read the Full Announcement Here

U.S. Senate passes the Bipartisan "NATIONAL ATAXIA AWARENESS DAY OF 2020" resolution

FOR IMMEDIATE RELEASE

ATAXIA PATIENT ADVOCACY ORGANIZATIONS APPLAUD THE U.S. SENATE FOR PASSING THE BIPARTISAN "NATIONAL ATAXIA AWARENESS DAY OF 2020" RESOLUTION


Can't see this document? Click Here

The inaugural International Congress for Ataxia Research (ICAR)


ICAR to take place March 15th – 18th, 2022

For Immediate Release

Downingtown, PA – (November 18, 2020) - The National Ataxia Foundation (NAF), the Friedreich’s Ataxia Research Alliance (FARA), and Ataxia UK are pleased to announce the date for the inaugural International Congress for Ataxia Research (ICAR) to be held at the Caribe Royale resort in Orlando, Florida, USA on March 15th – 18th, 2022.

The Congress will combine the best of our organizations’ popular research meetings, including NAF’s Ataxia Investigators Meeting (AIM) and the International Ataxia Research Conference (IARC), co-led by FARA, Ataxia UK, GoFAR, and other partner organizations. ICAR will cover aspects of cerebellar function, neurodegeneration, and treatment of ataxias, and will provide ample opportunities to network, as well as interact with ataxia patients and families. Leading the scientific steering committee are Co-Chairs Stefan-M. Pulst, M.D., Dr. med., Professor and Chair of the Department of Neurology at the University of Utah, USA, and Massimo Pandolfo, M.D., F.A.A.N., F.E.A.N., Professor of Neurology at Université Libre de Bruxelles, Hôpital Erasme, Belgium.

"I am extremely excited to co-chair this new ataxia meeting that will bring together clinicians and scientists from around the world and at all stages of their careers", says Professor Pulst. "The congress will cover diverse topics ranging from cerebellar function and imaging to novel therapies in sporadic and genetic ataxias".

As research on Ataxia continues to advance and pharmaceutical interest grows, international collaboration among patient advocacy groups, scientific and clinical researchers, and pharmaceutical companies is more critical than ever.

Professor Pandolfo states, “It is a great privilege to co-chair ICAR 2022, I expect exciting new developments in diagnosis, pathophysiology and particularly treatment. The meeting will be designed in order to maximize interaction, develop existing collaborations and prompt new ones.”

NAF, FARA and Ataxia UK are thrilled to be working together to host what will undoubtedly be hailed as the conference to attend in 2022! ICAR information will be available at ataxiacongress.org.

About NAF: NAF is a nonprofit organization established in 1957 to help persons with Ataxia and their families. The Foundation’s primary purpose is to support Ataxia research, provide vital programs and services for Ataxia families, and help in the search for a cure. NAF is the only organization in the United States dedicated to the disease that serves all types of Ataxia. NAF works closely with the world’s leading Ataxia researchers, promoting exchanges of ideas and innovation in Ataxia discovery.

About FARA: FARA in a non-profit, tax-exempt organization dedicated to the pursuit of scientific research leading to treatments and a cure for Friedreich’s ataxia. FARA's Mission is to marshal and focus the resources and relationships needed to cure FA by raising funds for research, promoting public awareness, and aligning scientists, patients, clinicians, government agencies, pharmaceutical companies and other organizations dedicated to curing FA and related diseases.

About Ataxia UK: Ataxia UK is the leading national charity in the UK supporting people affected by a range of ataxias. The charity actively promotes, facilitates and funds research into finding treatments and cures, and works collaboratively with numerous partners to drive research forwards. Ataxia UK also offers advice, information and support services to people affected by the ataxias.

NAF Executive Director:
Andrew Rosen
andrew@ataxia.org
763-553-0020
 
FARA Chief Executive Officer
Jennifer Farmer
Jen.Farmer@curefa.org
484-897-6160
 
Ataxia UK - Chief Executive:
Sue Millman
smillman@ataxia.org.uk
020-7582-1444
Media Contact:
Stephanie Lucas
Communications Manager, NAF
Stephanie@ataxia.org
763-231-2744

 
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