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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.

 

Girl tells author of ‘Wonder’ how book hit close and closer to home

Claire Juip meets R.J. Palacio as a winner of Letters About Literature contest.
Fifth-grader Claire Juip meets R.J. Palacio as a winner of Letters About Literature contest.
R.J. Palacio’s book “Wonder” has inspired millions of kids. It’s the story of a disfigured boy named Auggie who attends school outside his home for the first time as a fifth-grader. And it has prompted many discussions about courage, friendship and choosing to be kind.

For reader Claire Juip of Grosse Pointe, Michigan, the book’s message didn’t hit home the first time she read it. But two life-changing events caused Claire to reread it and reflect on it for the Library of Congress’s 2017 Letters About Literature contest.

The first event was finding out that her older brother, Jake, was diagnosed with a rare genetic disorder called Friedreich’s ataxia (FA).

Read the full article HERE

Friedreich’s Ataxia “Voice of the Patient” report

FARA is proud to release the Friedreich’s Ataxia “Voice of the Patient” report; a unique summary of direct testimony and survey responses from individuals living with Friedreich’s Ataxia (FA). This report is based on the proceedings surrounding the Externally Led Patient Focused Drug Development Meeting held on June 2, 2017 in Hyattsville, MD. The goal of the meeting was to give FA patients, and their families, friends and caregivers a forum to discuss their perspectives on living with the disease: what symptoms patients experience, how it affects their lives, what concerns them about the disease, their opinions on current and future treatment options, and experiences with research studies and clinical trials.

This meeting, and the report, represent important accomplishments and milestones for our community.

  • This was the largest FA meeting ever held with more than 400 people participating in person or online; including >20 FDA representatives, 25 drug company representatives and >350 FA patient and family participants.
  • It was the first time FA patients and other members of the FA community had an opportunity to communicate directly with representatives of the Food and Drug Administration.
  • Important themes and insights emerged that can inform drug development and regulatory decision making, such as the conclusion that “while the majority of patients noted that balance, dexterity and fatigue affected their lives the most now, most patients’ greatest concern for their future is the development/ progression of cardiomyopathy in the future, because it is life shortening”.

This document faithfully summarizes the testimony, comments and survey responses shared by individuals living with FA and their family members and caregivers.

We are very grateful to FDA for giving us the opportunity to hold this meeting, and for taking the time to participate and listen to our community. We hope that regulators and companies developing therapies for Friedreich’s ataxia will read this report and better understand the great unmet medical need of FA, the impact it has on patients and what patients seek in terms of treatment. This, in turn, will help companies develop therapies that affect the aspects of disease most important to patients, and regulators to approve those therapies with the greatest impact on patients.

We are grateful to all the individuals in the FA Community who participated in this meeting through providing testimony (both oral and written), attending the meeting, watching online and/or responding to the survey questions. We believe that your engagement in this process provided huge insight into the disease ,which will help the drug development community develop effective therapies.

The report can be read at  http://curefa.org/pdf/news/FA-Voice-of-the-Patient.pdf

A recording of the meeting in its entirety can be found at  https://www.youtube.com/watch?v=Va1D4SqrSfw
 


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10th Annual Friedreich’s Ataxia Symposium at CHOP

CHOP FA Sympoium 2017

For more information about the Symposium, please Click Here.

For registration information, please Click Here.

Fitchburg lawyer battling rare disease: ‘I want to walk’

Nick Carbone
It is a rare, inherited, progressive disease affecting one in 50,000 that results in loss of ambulation six to eight years after diagnosis and risk of premature death, with an average lifespan for those who have it of 35 years old.

Local attorney and politician Nicholas J. Carbone was diagnosed with Friedreich’s ataxia at 12 — when he was still able to walk. He is now 30.

Mr. Carbone is one of 10 people living with Friedreich’s ataxia chosen from across the country, and the only one from Massachusetts, to testify at a recent meeting of the U.S. Food and Drug Administration in Washington, D.C., attended by more than 25 FDA officials, more than 25 medical researchers and those from pharma, and 140 patients and family members.

Read more HERE

Student battling rare disorder — with a smile — wins national writing contest

Claire Juip
A Grosse Pointe fourth-grader's experience with a rare disease inspired her to write a letter that won first place in a national writing contest.

And Claire Juip, 9, is herself an inspiration — to her family, to her friends and to the community around her school, St. Paul Catholic School in Grosse Pointe Farms.

She and her brother, 11-year-old Jake, have Friedreich's ataxia. The genetic condition causes progressive damage to the parts of the nervous system involved in muscle control.

Despite the prospect of likely having to use a wheelchair someday, both children are filled with optimism, working to educate people about the condition and participating in sometimes painful medical studies in the hope of helping others.

Read more HERE

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