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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.

 

CANADA- FIRST TO APPROVE DRUG IN FRIEDREICH’S ATAXIA

The Muscular Dystrophy Association (MDA) and the Friedreich's ataxia Research Alliance (FARA) announced this week that, on July 24, the antioxidant compound idebenone received conditional market approval for the treatment of Friedreich's ataxia (FA) in Canada but failed to obtain such approval in Europe.

Idebenone, which is related to the natural substance coenzyme Q10, was developed by the Swiss pharmaceutical company Santhera as SNT-MC17.

The company is testing the compound, which is intended to improve cellular energy production and reduce oxidative stress, in FA, Duchenne muscular dystrophy (DMD) and Leber’s hereditary optic neuropathy.

In a phase 2 study in patients with FA, idebenone was generally well tolerated and associated with improvement in neurological function, as well as scores measuring activities of daily living.

A phase 3 trial in FA remains open.

Read more: CANADA- FIRST TO APPROVE DRUG IN FRIEDREICH’S ATAXIA

First Human Dose Initiated of Promising New Friedreich’s Ataxia Drug Highlights Effectiveness of New Public-Private Partnership Model

The Friedreich’s Ataxia Research Alliance (FARA) joins its public and private partners in announcing that a phase I clinical trial of a promising new drug, designated A0001, began dosing on July 11th. This milestone achievement illustrates the power of a new model for advancing therapies, especially in rare diseases.

“FARA believes in the essential nature of public-private partnerships that involve government agencies such as the National Institutes of Health (NIH) and the Food and Drug Administration (FDA), academic investigators, the pharmaceutical industry, and multiple non-profits like FARA and the Muscular Dystrophy Association (MDA),” said FARA President Ron Bartek. “FARA is grateful to its partners that span the spectrum from discovery through clinical development as we work together to advance promising compounds like A0001 toward the goal of approved treatments and cures for Friedreich’s Ataxia (FA) and other neurodegenerative disorders.”

Read more: First Human Dose Initiated of Promising New Friedreich’s Ataxia Drug Highlights Effectiveness of...

Request for Applications

Mitochondrial Therapeutic Approaches Research Award sponsored by Friedreich’s Ataxia Research Alliance and the Muscular Dystrophy Association

The Friedreich’s Ataxia Research Alliance (FARA) an d the Muscular Dystrophy Association (MDA) invite proposals, under a competitive Request for Applications (RFA) process, to award a grant focusing on critical issues or hypotheses that advance therapeutic approaches to mitochondrial dysfunction as it relat es to neurodegenerative diseases. We anticipate funding one award for two years under this program. The total award is limited to $100,000 (indirect costs can not exceed 10%) per year for two years.

Read More: Request for Applications

Congressman Andrews FA Awareness Day Resolution

Friedreich's Ataxia Awareness Day — (Extension of Remarks - May 13, 2008) — Speech of Hon. Robert E. Andrews of New Jersey in the House of Representatives Tuesday, May 13, 2008

Mr. ANDREWS. Madam Speaker, I rise today in support of Friedreich's Ataxia Awareness Day, which is recognized each year on the third Saturday in May. Friedreich's ataxia is a life-shortening neurological disorder that is usually diagnosed in childhood. It causes muscle weakness and loss of coordination in the arms and legs; impairment of vision, hearing and speech; scoliosis, diabetes; and a life-threatening heart condition. Most patients need a wheelchair full-time by their twenties. Life expectancy is reduced to early adulthood. There is currently no effective treatment or cure for Friedreich's ataxia.

Read more: Congressman Andrews FA Awareness Day Resolution

The Cake Eaters

Friedrich's ataxia, a progressive neurological disease that causes loss of muscle control, has entered the spotlight of the independent film world.

The Cake Eaters features as one of its central characters 16-year-old Georgia, who has Friedrich's ataxia. She is portrayed brilliantly by the young actress Kristen Stewart, who captures the disease's characteristic rag-doll weakness and awkward gait while projecting a smoldering adolescent sexuality. First-time director Mary Stuart Masterson, best known for her nuanced acting in Fried Green Tomatoes and Benny and Joon, videotaped interviews with real teenagers with Friedrich's ataxia to help prepare Stewart for the role.

But this slow moving and insightful film refuses to buy into the after-school special narrative of the heroic teen who overcomes her disability. Georgia's disability will not be overcome. The disease will persist until, as she puts it, “my heart gives out, and I don't know when that will be.” And Georgia is not always heroic. Her quest to seduce Beagle, a school cafeteria worker played with depth and intelligence by Aaron Stanford, at first barely acknowledges his humanity. She wants to know what sex is like, and he is the means to that end.

Read More: The Cake Eaters

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