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FARAFARA Cure FA

 

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.

 

JOIN AN UPCOMING LIVE WEBINAR TO LEARN ABOUT THE PATIENT FOCUSED DRUG DEVELOPMENT MEETING

JOIN AN UPCOMING LIVE WEBINAR on April 4th or April 11th
TO LEARN ABOUT HOW THE FA COMMUNITY CAN PARTICIPATE IN THE
PATIENT FOCUSED DRUG DEVELOPMENT MEETING
WITH THE FOOD AND DRUG ADMINISTRATION

The Friedreich’s Ataxia Research Alliance (FARA), in partnership with the Muscular Dystrophy Association and the National Ataxia Foundation, will be hosting two live webinars in April to discuss how FA patients and caregivers can participate in the very important, upcoming Patient Focused Drug Development meeting with the U.S. Food and Drug Administration (FDA) in June.

Please join us for one of the two live webinars (click "Read More" below) to view key information, including background information about the FDA & drug development, an overview of FDA Patient-Focused Drug Development, discussion questions & format, tips for participating in the meeting, and meeting logistics.
 

Rare Disease Week on Capital Hill, February 27 - March 2, 2017

Rare Disease Week
Ron Bartek and Brigid Brennan joined rare disease advocates from 48 states for the annual Rare Disease Week in Washington DC. The theme this year was research - a mission FARA has been supporting for almost 20 years. Here are some highlights:

Monday - Rare Disease Day at the National Institute of Health (NIH)

It was standing room only as doctors and advocates shared updates from their respective fields. Over 700 people were present and, for the first time, many more livestreamed from home. Everyone was in agreement that research is needed in the rare disease community with less than 500 treatments for over 7000 diseases. However, we are not alone in benefitting from goal. Rare disease are often the "window" into common disease treatments as well. Rep Leonard Lance (R-NJ) spoke about his continuing support for the rare disease community. The crowded erupted in applause to learn that Dr Francis Collins, a champion for our community, was asked to stay on as head of the NIH.


That evening Rare Disease Legislative Advocates (RDLA) hosted a cocktail reception and documentary screening of Up for Air. Senator Ed Markey (D-MA) and Representatives Jim McGovern (D-MA) made brief remarks of support. Our own Ron Bartek moderated the panel discussion of the movie where everyone left in awe by Jerry Cahill's inspirational journey with Cystic Fibrosis.
 

FA Patient Focused Drug Development Meeting - Your opportunity to tell the FDA what is important to you in finding a treatment for Friedreich’s Ataxia

What is a Patient Focused Drug Development Meeting?

In 2015 the Food and Drug Administration (FDA) began a new initiative to expand the way it looks at therapies, specifically seeking input from patients. The agency wanted to hear directly from the community about patients’ needs, rather than assuming that internal FDA scientists understood every disease and patient population. Thus, it launched the Patient-Focused Drug Development (PFDD) Initiative, a series of public meetings led by the FDA and designed to systematically gather input from patients regarding their experience of living with specific diseases, the preferred impact of potential treatments, and the benefit/risk analysis when considering a new targeted medication.. The FDA scheduled the initial 20 meetings, many of which have taken place and are considered very successful. In order to reduce their administrative burden however, the FDA, has now moved to a new model where such meetings are led by patients and patient groups, with the FDA attending and participating. FA has been selected to be one of the disease areas to host such a meeting in 2017.

Continue reading HERE.

Get Involved: Friedreich’s Ataxia Patient Focused Drug Development (PFDD) Meeting

news02202017
Get Involved: Friedreich’s Ataxia Patient Focused Drug Development (PFDD) Meeting.
Tell the FDA what is important to you in finding a treatment for Friedreich’s Ataxia

 
To learn more about the PFDD meeting and how you can contribute, click HERE.
 
An upcoming Friedreich’s ataxia (FA) Patient Focused Drug Development (PFDD) meeting with the U.S. Food and Drug Administration (FDA) is your opportunity to tell FDA and drug developers about challenges and burdens you have experienced with FA, and share your thoughts about what is most important to you in evaluating potential new treatments for the disease.
 
The meeting, co-organized by the Friedreich’s Ataxia Research Alliance, Muscular Dystrophy Association and National Ataxia Foundation, marks the first time patients and families affected by FA will be able to speak directly to the FDA and share their experiences in their own words.
 
Information captured at the meeting, summarizing input about the patient experience from people with FA across the country, will be published in a “Voice of the Patient” report and submitted to the FDA for inclusion in the framework used to evaluate future FA therapies.
 
There are several ways you can get involved:

  • Attend the PDFF meeting in Bethesda, Md. on June 2, from 8 a.m. – 12:30 p.m., at the College Park Marriott and Conference Center.
  • If you cannot attend in-person, join online via streaming webcast and share your input on the specific panel questions, as well as demographic questions.
  • Keep an eye out for future communications and surveys through which you may be able to contribute your thoughts.

No one can make the voice of the FA community heard more than those impacted by the disease. Your participation is critical to making sure our collective voice makes an impact. Don’t miss out on the opportunity to make sure your input helps guide the development of successful, effective, meaningful treatments for FA.

2nd International Ataxia Research Conference - September 27-30, 2017 - Pisa, Italy

The 2nd International Ataxia Research Conference will be held on September 27-30, 2017 in Pisa, Italy. The focus of the meeting is a comprehensive scientific review of new research from disease definition to therapeutic treatments. The conference will include Friedreich’s ataxia and other recessive ataxias (eg: ataxia with oculomotor apraxia), dominant ataxias (eg: spinocerebellar ataxias, DRPLA, episodic ataxias) and autoimmune ataxias.

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