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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.

 

Penwest Pharmaceuticals (PPCO) Announces Results of Phase Ib Trial of A0001

Penwest Pharmaceuticals Co. (Nasdaq: PPCO) announced the results of its Phase Ib clinical trial of A0001, a compound the Company is developing for the treatment of mitochondrial diseases. Drs. Amale Hawi, Senior Vice President, Pharmaceutical Development, and Thomas Sciascia, Senior Vice President & Chief Medical Officer, will be presenting these results at the Friedreich's Ataxia Therapeutics Meeting on Friday, July 17, 2009, in Philadelphia, PA.

In the Phase Ib trial, the drug was well tolerated by subjects, and no serious adverse events were reported. There was a dose-dependent increase in exposure approaching steady state within 2-4 days following repeat-dosing, and a maximum tolerated dose was established.

Read More: Penwest Pharmaceuticals (PPCO) Announces Results of Phase Ib Trial of A0001

2009 Kyle Bryant Translational Research Award sponsored by Ride Ataxia, the Friedreich’s Ataxia Research Alliance and the National Ataxia Foundation

The Friedreich's Ataxia Research Alliance (FARA) and the National Ataxia Foundation (NAF) invite proposals, under a competitive Request for Applications (RFA) process, to award grants focusing on pre-clinical and clinical investigations that will advance treatments for Friedreich's Ataxia. FARA and NAF are grateful to the Ride Ataxia cyclist s and supporters as well as sponsor Outback Steakhouse who collectively raised $260,000 .

FARA and NAF will provide matching funds to bring the grant award total to $360,000. As a result a total of three awards will be funded under this program, each limited to $120,000 (direct costs only). Two awards will be funded by FARA and NAF, and the third award will be funded by FARA made possible by the sponsorship from Outback Steak house. Proposals should be for pre-clinical / translational or clinical research focused on Friedreich's ataxia with aims such as identification of biomarkers for FA, development of animal or cellular models to evaluate candidate therapies, development of tools or technologies for therapy development, pre-clinical development and testing of potential targets, or clinical studies of patient outcome measures.

Read More: 2009 Kyle Bryant Translational Research Award sponsored by Ride Ataxia, the Friedreich’s Ataxia Research Alliance and the National Ataxia Foundation

Fitness Together Helps Client in Wheelchair Fight Disease with Exercise

When Kerry Westfall was diagnosed at age 16 with a rare neurodegenerative disease that had no treatments or cures, the news was devastating. But 15 years later with the debut of his own exercise DVD, he couldn’t be more optimistic.

That’s because, at 31, Westfall did the unexpected when he decided to videotape himself exercising. Confined to a wheelchair with the genetic disorder Friedreich’s ataxia, Westfall was faced with a future of muscle weakness, a loss of arm and leg coordination , curvature of the spine, vision impairment, hearing loss, slurred speech and early death. He overcame t he depression of his situation and got active inste ad. With the help of personal trainer Ed Ferrell, who owns a Fitness Together studio in Temecula, Calif., the two put together a fitness schedule that has helped Westfall maintain maximum control over his body in spite of his medical condition.

Read More: Fitness Together Helps Client in Wheelchair Fight Disease with Exercise

New study to examine whether smoking cessation drug Varenicline may benefit adults with Friedreich’s ataxia

A drug approved for smoking cessation may hold promise for people suffering from Friedreich’s Ataxia (FA), an inherited disease that causes progressive damage to the neuromuscular system. A new clinical study, sponsored by the Friedreich’s Ataxia Research Alliance (FARA), will investigate whether varenicline (Chantix®) improves neurological symptoms, such as balance, coordination, and sensory perception, all of which are significantly impaired in patients with FA. Chantix® is approved by the U.S. F ood and Drug Administration to help cigarette smokers stop smoking. Currently, there is no effective treatment for FA.

The double blind, randomized, placebo-controlled pilot study will be led by principal investigator Dr.Theresa Zesiewicz, professor of neurology at the University of South Florida College of Medicine, and co-investigator Dr . David Lynch, associate professor of neurology and pediatrics at Children’s Hospital of Philadelphia. Dr. Zesiewicz noticed that the uncoordinated movements (ataxia) and balance problems of a patient with fragile X tremor /ataxia syndrome imp roved greatly after he started varenicline in an attempt to quit smoking. The symptoms worsened when the medication was discontinued. Dr. Zesiewicz found similar results when treating patients with other types of ataxia, and several of her case reports were published last year in medical journals.

Read More:New study to examine whether smoking cessation drug Varenicline may benefit adults with Friedreich’s ataxia

Santhera's US Phase III IONIA Trial in Friedreich's Ataxia Misses Primary Endpoint

Data from second Phase III study expected in the first half of 2010
 
Santhera Pharmaceuticals (SIX: SANN), a Swiss specialty pharmaceutical company focused on orphan neuromuscular diseases, announced today that its US Phase III clinical trial evaluating Catena® for the treatment of Friedreich's Ataxia missed its primary endpoint as measured by the International Cooperative Ataxia Rating Scale (ICARS). The study also did not show statistical significance on the second neurological endpoint, the Friedreich's Ataxia Rating Scale (FARS). On both endpoints, the active treatment arms showed a consistent improvement over baseline and placebo, as seen in prior studies. However, due to a lower than expected effect size combined with the fact that patients on placebo improved unexpectedly, statistical significance could not be achieved in this study population. The safety results were consistent with published data suggesting that Catena® is safe and well tolerated at doses up to 2250 mg/day.

Read More: Santhera's US Phase III IONIA Trial in Friedreich's Ataxia Misses Primary Endpoint

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