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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.

 

Former teacher continues to beat the odds

“I would like to see a treatment or cure in my lifetime. Not only is that my Christmas wish; it’s my wish everyday.”
Christin Haun, diagnosed in 1985 with Friedreich’s Ataxia

It’s difficult to know what one might notice first about Christin Haun — her beautiful face, captivating smile or contagious sense of humor. She laughs easily and often.

It is a surprise then to hear her story and know at the emotional age of 15, this typical teenager was told she would be in a wheelchair by 20 and dead by 30.

“There were some confusing changes,” Haun said, “And, I had no idea why.” Her mother took her to a neurologist who offered devastating news about what lay ahead.

Read the entire article HERE

Gavin Lambert, 10, doesn't allow disorder to hold him back


When Gavin Lambert was 5 years old, his parents, Kevin and Dawn Lambert, learned that he had Friedreich's ataxia, a degenerative and debilitating neuro-muscular disorder that impairs basic abilities such as walking, hearing, vision and movement.

Read the entire article HERE

Ron Bartek receives Lifetime Achievement Award

Ron Bartek receives Abbey Award
Congratulations to FARA's Founder and President Ron Bartek on receipt of "The Abbey"- an award for Lifetime Achievement for the Rare Disease Community. Ron was honored in the Patient Advocacy category and received the award at the RareVoice Awards Gala hosted by the Rare Disease Legislative Advocates. He is pictured here with Dr. Stephen Groft who was also honored with the same award in the Government Agency category for his dedication to stimulating research and advancing development of therapies during his tenure at the Food and Drug Administration (FDA) and National Institutes of Health (NIH). Congressman Fred Upton, sponsor/author of the 21st Century Cures Act, also received the Lifetime Achievement Award in the Member of Congress category.

Read the entire article HERE

Testing the FA-ITH Four Years Later

Matt Rupel

“I have to say, Friedreich's ataxia (FA) is kicking my butt.” That single line brought everyone back to the reality of why they were gathered together on Saturday night in the Sunnyvale Community Center Ballroom for the Friedreich's ataxia In The Heart (FA-ITH) fundraiser. The fun “in a fundraiser” was at the forefront as guests wined and dined and enjoyed the evening. But the main reason everyone was gathered was to help raise money to fight a dreadful disease that is having terrible consequences on a close friend. The close friend is Matt Rupel.

Read the entire article HERE

UAB Researchers Receive Funding to Seek Biomarkers in Friedreich's Ataxia

The University of Alabama at Birmingham researchers have received funding to seek and validate biomarkers for Friedreich's Ataxia with a two-year, $140,000-a-year grant funded by The Friedreich's Ataxia Research Alliance (FARA) and FARA Ireland.

Discovery of validated biomarkers is a high priority because they are invaluable to help speed the development and clinical evaluation of potential drugs. FARA's treatment pipeline already includes nine potential drugs or treatments that are in human clinical trials, seven more that are in preclinical testing, and four others in the early discovery phase.

Marek Napierala, Ph.D., assistant professor in the UAB Department of Biochemistry and Molecular Genetics, UAB Stem Cell Institute, and Jill Butler, Ph.D., instructor in Biochemistry and Molecular Genetics, are co-investigators. ‪

In preliminary work for the grant application, Butler and Napierala looked at RNA gene expression in patient and control fibroblast cells. They identified several gene expression variants as candidate biomarkers, and they now are working to validate those candidates in neuronal and cardiac Friedreich’s ataxia cell line models and to adapt these biomarkers for routine testing in a clinical setting. They measure protein expression levels using reverse phase protein arrays, and they have future plans to look for changes in microRNAs in the Friedreich’s ataxia cells. MicroRNAs are short sequences of RNA that act as robust agents of gene regulation in cells.

“For this research to be successful and make it to the clinic,” Napierala said, “the test or measurement needs to be easily done.” Ideal biomarkers also have to be safe, cost effective and easily adapted from research laboratory testing to the clinic.

Read the entire article HERE

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