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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.

 

Tell Congress to Keep the Orphan Drug Tax Credit!

Dear FA Ambassadors / American FA families,

As we all know, the US Congress is currently working on tax reform. One of the current laws being reviewed is the Orphan Drug Tax Credit which was enacted as part of the Orphan Drug Act in 1983. This law is very beneficial to the FA community. It has successfully encouraged drug development in rare disease and we do not want to lose this incentive for research and therapy development.

NORD details the concern beautifully in their policy alert below and provides the links to enable you to contact your two Senators and House of Representative member. Phone calls are given more attention in government offices but if time or comfort does not permit a call, please join the effort by sending an email to your three representatives. On the NORD Alert, when you click on “emailing your senators” and enter your address, you are provided the text for your email, You can personalize the email message if you wish and type in your name at the bottom. When you click on “submit,” the email is sent to your two senators and one house representative. So, this important action should take only a few minutes.

All senators and house members will be important in the final vote and some will have more influence over what happens based on the committees on which they sit. If one of your senators or your house representative is among those listed below, your voice could be the one that sways their vote to support the Orphan Drug Tax Credit. Please contact them as soon as possible and urge them to vote to preserve this important incentive to treat and cure FA and other rare diseases.

Warm regards,
Ron Bartek & Brigid Brennan

US Senate
Committee on Finance


Chairman Orrin Hatch (UT)
Ranking Member Ron Wyden (OR)

Chuck Grassley (IA)
Mike Crapo (ID)
Pat Roberts (KS)
Michael Enzi (WY)
John Cornyn (TX)
John Thune (SD)
Richard Burr (NC)
Johnny Isakson (GA)
Rob Portman (OH)
Patrick Toomey (PA)
Dean Heller (NV)
Tim Scott (SC)
Bill Cassidy (LA)
Debbie Stabenow (MI)
Maria Cantrell (WA)
Bill Nelson (FL)
Roberty Menendez (NJ)
Thomas Carper (DE)
Benjamin Cardin (MD)
Sherrod Brown (OH)
Michael Bennet (CO)
Robert Casey (PA)
Mark Warner (VA)
Claire McCaskill (MO)















House of Representatives
Ways and Means Committee


Chairman Kevin Brady (TX)
Ranking Member Richard Neal (MA)

Sander Levin (MI)
John Lewis (GA)
Sam Johnson (TX)
Lloyd Dogged (TX)
Devin Nunes (CA)
Mike Thompson (CA)
Pat Tiberi (OH)
John Larson (CT)
Dave Reichert (WA)
Earl Blumenauer (OR)
Peter Roskam (IL)
Ron Kind (WI)
Vern Buchanan (FL)
Bill Pascrell (NJ)
Adrian Smith (NE)
Joseph Crowley (NY)
Lynn Jenkins (KS)
Danny Davis (IL)
Erik Paulsen (MN)
Linda Sanchez (CA)
Kenny Marchant (TX)
Brian Higgins (NY)
Diane Black (TN)
Terri Sewell (AL)
Tom Reed (NY)
Suzan DelBene (WA)
Mike Kelly (PA)
Judy Chu (CA)
Jim Renacci (OH)
Pat Meehan (PA)
Kristi Noem (SD)
George Holding (NC)
Jason Smith (MO)
Tom Rice (SC)
David Schweikert (AZ)
Jackie Walorski (IN)
Carlos Curbelo (FL)
Mike Bishop (MI)


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Friedreich’s Ataxia Community Stays on Top of Science and Stays in Touch

Patients and families dealing with the rare, debilitating disease Friedreich's Ataxia showed their true strength at the 10th Annual Friedreich's Ataxia Symposium held in King of Prussia, Pa. The FA community is a steadfast supporter of research, and they have been gathering for a decade to spend a day with each other building friendships and learning the most up-to-date information on the therapeutic approaches and scientific studies being conducted in the field of FA.

Researchers from Children's Hospital of Philadelphia took a moment during each of their presentations to sincerely thank the attendees: "We could not make these discoveries without you."

Read more HERE

FARA Announces Recipients of the 2017 Ataxian Athlete Initiative (AAI)

DOWNINGTON, PA-- The Friedreich's Ataxia Research Alliance (FARA) is proud to announce the recipients of this year's Ataxian Athlete Initiative (AAI). The AAI is a unique program that provides adaptive cycling equipment to people with ataxia who have demonstrated a strong desire to stay healthy and fit despite their progressive disabilities.

Friedreich's ataxia (FA) is a rare disease that affects balance and coordination and has symptoms such as vision and hearing loss and often results in life shortening heart complications.

This year, the AAI was sponsored by UVA Sun Systems, The Melting Pot, Catrike, The FA Project, The Texas Irish Foundation and The Burrows Hill Foundation. The Burrows Hill Foundation generously provided over 70% of the funding for the 2017 AAI.

"The Burrows Hill Foundation is so proud to team with such a worthy and well established program such as the AAI. We are aware how important it is for those with ataxia to stay active and healthy and are honored to be able to help provide adaptive cycling equipment to these worthy applicants."

Joni Moore
Joni Moore shares her story HERE

AAI grants are administered through a competitive application process. Applicants research the most appropriate adaptive cycling equipment to suit their abilities and describe how such equipment would help them to reach their fitness goals and improve their quality of life.

"I am in love with riding my new handcycle, receiving it has honestly changed my life," said 2017 grant recipient Natchez Hansen. "I am building muscles and getting in shape for Orlando’s Ride Ataxia event November 12."

The AAI has provided equipment for 42 individuals since 2009. In 2017, the AAI funded 9 grants for adaptive cycling equipment to athletes from 7 states and 2 countries including:
Ashlea Smith of Aliquippa, PA,
Jacob Tompkins of Tivoli, NY,
Joni Moore of Superior, WI,
Justin Hernandez of Graham, TX,
Katie Hook of LaPorte, IN,
Kevin Henry of Jackson, MO,
Natchez Hanson of Palm Coast, Fl
and Savvas Nicolaou of Limasoll, Cypress

Visit http://curefa.org/aai for more information and to view more photos of past recipients. The next application cycle begins in Spring 2018.

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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