PTC Therapeutics will be hosting an Informational Patient Webinar on July 28th at 7pm for those interested in learning more about PTC-743, a candidate molecule for the treatment of Friedreich's ataxia. The clinical trial for PTC-743 will be international and include clinic sites in Australia, Brazil, Canada, the E.U., and the U.S.

Please email info@curefa.org if you are interested in attending this Webinar.

Hello friends,

As we move into summer and the country works through a phased reopening, academic and clinical researchers are coming back online with social distancing measures in place. For many academic researchers, this means lab activities beginning to resume but not at full capacity, and many lab teams working in staggered schedules to reduce the number of staff in a space at any one time.

Our Collaborative Clinical Research Network (CCRN) sites are beginning to see patients in person again for both clinical and research visits. During the first months, they will continue to work at a reduced volume and with new practices and procedures in place to protect staff and patients. We encourage you to contact your site to learn how best to proceed. Whether you are involved in ongoing research or considering participating in a new study, your coordinator can discuss options for in-person versus telemedicine visits.

Thank you for your ongoing support of FA research ...

Read the Full Monthly Update

Hello friends,

As we move from spring to summer and parts of the country begin their phased reopening, I wanted to update you on the status of academic and clinical research in FA. Throughout the time of closures due to COVID-19, we have remained in touch with FARA funded academic and clinical researchers to understand their challenges and offer support.

Currently, both academic and clinical researchers are starting to come back online with social distancing measures in place. For many academic researchers, this means lab activities beginning to resume but not at full capacity. We anticipate that many lab teams will be working in staggered schedules to reduce the number of staff in a space at any one time.

Additionally, our Collaborative Clinical Research Network (CCRN) sites are beginning to see patients in person again for both clinical and research visits. This will be at reduced volume and with new practices and procedures in place to protect staff and patients. We encourage you to contact your site to learn how best to proceed. Whether you are involved in ongoing research or would like to consider participating in a new study, your coordinator can discuss options for in-person versus telemedicine visits. For studies that require in-person visits, some topics you may wish to discuss are:

1. What are the onsite measures in place to keep staff and patients safe during in-person visits?
2. Can I wear my own personal protective equipmet (PPE) or will the study provide this?
3. Will I need to be tested for Covid-19 prior to a visit? If so, how will that be arranged?
4. Are there any location changes to where the study is being conducted?

Hello friends,

As you know, May is FA Awareness month. A special thanks to everyone who got involved in the campaigns, events and talks throughout the month. Members of the community flexed (at home) to raise funds through the social campaign #LendUsSomeMuscle, logged miles and fundraising dollars in the rideATAXIA NorCal Virtual Challenge and the Gehr Family hosted a live stream of awesome musical performances in this year's Cure FA Soirée. Pharma partners Exicure, PTC Therapeutics and Avexis all honored FA Awareness month by inviting FARA and FA patient panelists to their virtual team meetings. And FARA hosted our own flash-talk series featuring presentations from Young Investigators on their FA research.

There is always a way to remain active, even from home. One specific research need is for Parents of FA Children age 12 and under and Patients ages 13-17 to complete The Friedreich's Ataxia Health Index Study. This study aims to identify the symptoms that have the greatest impact on quality-of-life for individuals with FA. The results of the study will help guide future research involving FA patients through the development of a patient-reported measure of health. The study involves completing a survey to include questions on demographics and symptoms of FA. ...

Read the Full Monthly Update

The Friedreich’s Ataxia Research Alliance Hires Barbara Tate, Ph.D. as FARA’s New Chief Scientific Officer

Downingtown, PA (May 27, 2020) - The Board of Directors of the Friedreich’s Ataxia Research Alliance (FARA) today announced the hiring of Barbara Tate, Ph.D. to the full-time role of Chief Scientific Officer (CSO). FARA is a national, non-profit organization dedicated to the pursuit of scientific research leading to treatments and a cure for Friedreich’s ataxia (FA). Friedreich’s ataxia is a rare debilitating, life-shortening, degenerative neuro-muscular disorder. About 5,000 people in the United States and 15,000 worldwide live with FA.

Dr. Tate’s hiring marks the organization’s growing commitment of resources to FA research advancement on multiple fronts. As the Chief Scientific Officer, Dr. Tate will work closely with FARA’s staff, Board of Directors and Scientific Advisory Board to set and execute FARA’s research strategy and ensure a deep and diverse treatment pipeline with well-informed and vetted treatment approaches. Dr. Tate’s responsibilities will include the oversight of all research and drug development activities as well as leading FARA’s research staff. Dr. Tate will seek to build international engagement and collaboration with academic and industry scientists, to address gaps in drug development and explore novel answers to the most challenging FA research questions.

Dr. Tate comes to FARA with a strong background in the discovery and development of therapies for central nervous system diseases. Early in her career she was responsible for a portfolio of early stage neurodegenerative focused therapeutics at Pfizer. More recently, she has gained experience as a Venture Fund investor and Biotech Executive. Prior to her role at FARA, Dr. Tate held a leadership position at the Dementia Discovery Fund (DDF), a specialist venture capital fund investing in and creating companies to deliver high impact therapeutics for age-related dementias. Dr. Tate has also held leadership positions at Tiaki Therapeutics (Founding CEO), Rodin Therapeutics, an Atlas Venture Company, Armada Therapeutics and Satori Pharmaceuticals, and has served on the boards of several biotech companies.

FARA Chief Executive Officer Jennifer Farmer said, “We are excited to welcome Barbara to the FARA team. She understands the sense urgency felt by people with progressive neurological conditions and unmet medical need, and she is committed to the strategic leadership required to accelerate and expand our research efforts to meet that need. Barbara brings significant experience in neuroscience, drug development and life science investing, all of which she will direct at our mission as we grow and advance our research programs and treatment pipeline.”

Dr. Tate said, “I am thrilled to become part of the FARA team. The passionate and forward-thinking FA community has laid down a solid foundation of scientific insights, patient data, and clinical trial expertise to drive toward meaningful therapeutics for this devastating disease. I feel privileged to be part of the next exciting chapter at FARA”.

The CSO role was previously held on a part-time basis by Dr. Giovanni Manfredi, Weill Cornell Medicine. FARA is grateful to Dr. Manfredi as he has provided rigorous scientific oversight and leadership to the organization’s programs since 2010, including his recognition of the need for full-time staff and assistance in identifying Dr. Tate for this critical role. Dr. Manfredi will remain involved with the organization in a volunteer capacity, serving as FARA’s Co-Chair of the Scientific Advisory Board and Scientific Review Committee.

Dr. Tate did her Postdoctoral Fellowship at Harvard Medical School and earned her Ph.D. at the University of Delaware.

View the PDF