A Call for FA Patients to Join Global Registry to Advance Knowledge, Treatments

A Call for FA Patients to Join Global Registry to Advance Knowledge, Treatments
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People with Friedreich’s ataxia (FA) are being urged to join the Friedreich’s Ataxia Global Patient Registry (FAGPR) to help advance knowledge into disease progression and support treatment development.

Managed by international advocacy organizations, the patient registry seeks to collect data from volunteers worldwide to improve clinical trial efficiency. A more robust registry would give the studies, and the prospective treatments they test, a better chance of succeeding. And, the FAGPR is a good way for FA families to stay abreast of research developments.

In addition, because the registry gathers demographic and disease information, the patient-entered data helps provide an idea of how common FA is in different parts of the world, what age groups are most affected, and how the disease affects quality of life. In turn, this could identify needs for additional research into the neuromuscular disorder, which affects 1 in every 50,000 people globally.

FAGPR also aims to characterize the spectrum of symptoms and heterogeneity of FA presentation — the likelihood of multiple patients having different symptoms — establish the rate of disease progression, and enable swift clinical trial launches through informing eligible patients.

“When pharmaceutical companies begin the journey of developing a drug for FA, one of the first questions asked is, ‘Where are FA patients located around the world?’” the FAGPR website states.

Having answers allow these companies to plan for clinical trials and the eventual distribution of treatments once approved, and to be aware of regulatory approval processes in each country where FA patients are treated. Companies also need to plan for physician education about therapy availability in any country where FA patients are being diagnosed and treated.

The registry data does not contain individual patient contact information and is never shared with scientists or pharmaceutical companies without protocol review of how the information will be used.

After completing the consent process, participants can create a patient profile of demographic information, which can be updated at any time. Afterward, a series of surveys take between five and 20 minutes to complete. Those surveys can be updated each year. Go here to enroll.

Parents of minors diagnosed with FA can register on their behalf. For more information, contact the registry coordinators at [email protected]

The Friedreich’s Ataxia Research Alliance first opened a registry for the FA community in 2005. In 2019, the platform was updated to better accommodate the entire FA community and to comply with new privacy rules. The new system also is able to handle foreign language translation and more research capabilities.

To date, more than 830 patients from 51 countries have enrolled in the registry.

Mary M. Chapman began her professional career at United Press International, running both print and broadcast desks. She then became a Michigan correspondent for what is now Bloomberg BNA, where she mainly covered the automotive industry plus legal, tax and regulatory issues. A member of the Automotive Press Association and one of a relatively small number of women on the car beat, Chapman has discussed the automotive industry multiple times of National Public Radio, and in 2014 was selected as an honorary judge at the prestigious Cobble Beach Concours d’Elegance. She has written for numerous national outlets including Time, People, Al-Jazeera America, Fortune, Daily Beast, MSN.com, Newsweek, The Detroit News and Detroit Free Press. The winner of the Society of Professional Journalists award for outstanding reporting, Chapman has had dozens of articles in The New York Times, including two on the coveted front page. She has completed a manuscript about centenarian car enthusiast Margaret Dunning, titled “Belle of the Concours.”
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Inês holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she specialized in blood vessel biology, blood stem cells, and cancer. Before that, she studied Cell and Molecular Biology at Universidade Nova de Lisboa and worked as a research fellow at Faculdade de Ciências e Tecnologias and Instituto Gulbenkian de Ciência.
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Mary M. Chapman began her professional career at United Press International, running both print and broadcast desks. She then became a Michigan correspondent for what is now Bloomberg BNA, where she mainly covered the automotive industry plus legal, tax and regulatory issues. A member of the Automotive Press Association and one of a relatively small number of women on the car beat, Chapman has discussed the automotive industry multiple times of National Public Radio, and in 2014 was selected as an honorary judge at the prestigious Cobble Beach Concours d’Elegance. She has written for numerous national outlets including Time, People, Al-Jazeera America, Fortune, Daily Beast, MSN.com, Newsweek, The Detroit News and Detroit Free Press. The winner of the Society of Professional Journalists award for outstanding reporting, Chapman has had dozens of articles in The New York Times, including two on the coveted front page. She has completed a manuscript about centenarian car enthusiast Margaret Dunning, titled “Belle of the Concours.”
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