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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.


Monthly Update - January 2021


Dear friends,

As many of you know, FARA has prepared a letter to send to the Food and Drug Administration (FDA) and Reata Pharmaceuticals to request that they Allow Individuals with Friedreich Ataxia Access to Omaveloxolone.
Read and Sign on to the letter
This letter requests Reata to submit a New Drug Application (NDA) on an urgent basis and FDA to exercise the flexibility granted by law and contained in FDA guidance in considering approval of an NDA for Omaveloxolone in FA based on the existing evidence from clinical trials.

In just four days, you have facilitated over 25,000 signatures in support of this letter! We are grateful to everyone who has signed the letter, especially members of our FA families who make up 23% of respondents. The statements and photos shared by FA families will be instrumental in drawing attention to the patient experience and voice. If you have not signed onto the letter, you can do so up until January 20th. The entire FA community (individuals with FA, family, friends, caregivers, supporters, other rare disease advocates) is invited to sign on.

More...

Read the Full Monthly Update
 

Webinar: FA Community Call to Action on January 5, 2021

Webinar: FA Community Call to Action - held on January 5, 2021
The sign-on period will remain open until January 20, 2021 at 5pm (Eastern)
Have Your Voices Heard: Be a Part of the FA Community's Request for Access to Reata and FDA

Watch the Recorded Webinar

(See below for more detailed information about MOXIe)

After consultation with regulatory advisors, FARA plans to submit a letter to both Reata and the Food and Drug Administration (FDA) that requests they work together to provide access to omaveloxolone for individuals with Friedreich's Ataxia as soon as possible. The letter will highlight the voice of the patient with a request for Reata to submit a marketing application to the FDA and for the FDA to review and consider approval of this application. The strength of this letter will depend in large part on input and testimony from the FA community. The voices and lived experiences of this community matter and will help add critical perspective and context to the MOXIe trial data points that are being discussed. Even if you did not participate in this clinical trial, it is important to hear your voices too. It is crucial that our response is respectful, informative, and representative of the FA patient experience.

We are at an important crossroads in Reata's development of omaveloxolone as a potential treatment for FA. Our clinical investigators and FARA have confidence in the data from the completed studies and would like FDA to use tools given to it under the law to exercise regulatory flexibility to avoid a delay of at least several years for access which would occur if Reata conducts another clinical trial prior to the drug being approved for use by FA patients in the U.S. Please register for this important session and learn how you can participate. Please also share this message within the FA community. The greater our unity, the stronger our message.   more...

Read the Full Text
 

Friedreich's Ataxia Center of Excellence at CHOP Awarded $1.275 Million to Advance Medical Research

The Friedreich's Ataxia Center of Excellence (COE) at Children's Hospital of Philadelphia (CHOP) was awarded $1.275 million by the Friedreich's Ataxia Research Alliance (FARA), along with the Hamilton and Finneran families and the CureFA Foundation, to support the development of breakthrough therapies to improve the quality of life for individuals with Friedreich's ataxia.

Friedreich's ataxia (FA) is a neurodegenerative condition that limits the production of frataxin, a protein in the mitochondria, the cells' energy generators. The condition damages the nervous system and impairs muscle coordination, causing problems with movement and sensation that get progressively worse. Individuals may also experience cardiac dysfunction, scoliosis, diabetes, vision and hearing difficulties. About 1 in 50,000 individuals worldwide have FA, including 5,000 cases in the United States. Symptoms typically appear between age 5 and 15, though the condition can sometimes appear later in life as well.   more...

Read the Full Article
 

Monthly Update - December 2020


Dear friends,

As 2020 draws to a close, I reflect on the challenges, accomplishments, and continued generosity of our FA our community. One of the challenges we faced this year was the combination of increased research opportunities and fewer fundraising events.

This past Tuesday, aka Giving Tuesday, we launched our #GiftOfResearch campaign to help us close the funding gap and accelerate research momentum. As always, our community answered the call. We are excited to announce that Giving Tuesday kicked off our year-end campaign by raising over $95,000 which included a $25,00 generous match from Foley, Baron, Metzger & Juip, PLLC and a FARA Board Director.

Thank you to everyone that participated by creating an online fundraiser, donating, or by sharing photos and videos of why the #GiftOfResearch is important to you.

When you give the gift of research this holiday season, you are providing research tools and insights into Friedreich's ataxia. As you plan your holiday shopping, please consider giving the #GiftOfResearch to a loved one- gifting them an opportunity to be a part of the effort to find meaningful treatments and a cure for FA.

More...

Read the Full Monthly Update
 

Central Nervous System Therapeutic Targets in Friedreich Ataxia

Ian H. Harding, PhD, David R. Lynch, Arnulf H. Koeppen, and Massimo Pandolfo, MD
Published in Human Gene Therapy, 25 Nov 2020 https://www.liebertpub.com/doi/10.1089/hum.2020.264

Because many of the major targets of long-term therapy for FA are in the central nervous system (CNS), FARA worked with several FA investigators to write an in-depth review of the pathology present in the brain and spinal cords of patients with FA. The diverse group of experts was able to evaluate evidence gathered from human clinical observations, physiological, pathological and imaging approaches, as well as studies in animal models. The resulting paper provides detailed summary of the current understanding of what areas of the spinal cord and brain are impacted by the disease, including what specific cells are vulnerable to the loss of frataxin.

Moreover, the review also provides some insight into the temporal course of neuropathological changes in the CNS, which helps to provide information on areas that may respond to therapy and be successfully treated at different timepoints during the progression of the disease.

This comprehensive review of the impact of FA in the CNS is useful to patients, caregivers, physicians, as well as drug developers, especially those planning to test therapeutics aimed at the spinal cord and/or brain. FARA effort to organize and support production of this review represents the ongoing commitment to focusing resources on filling gaps in our understanding of FA and building relationships needed to find effective treatments.

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