The Friedreich’s Ataxia Research Alliance (FARA) has announced the 2016 winners of the Ataxian Athlete Initiative (AAI), a unique program providing adaptive cycling equipment to people with ataxia who have shown a strong commitment to staying healthy and fit despite their progressive illness. Since 2009, 33 people have been…
The Friedreich’s Ataxia Research Alliance (FARA) joined the California NAACP and other patient advocacy groups to listen to opinions and concerns about SB 1010 — a controversial California state bill that could potentially impact Friedreich’s ataxia (FA) care in the state. Those opposed to the bill, sponsored by state Sen. Ed Hernandez, claim it “prioritizes…
Jupiter Orphan Therapeutics Inc. (JOT) announced that David Sinclair, a Harvard Medical School professor with an interest in the dietary supplement resveratrol and its potential benefit in treating diseases such as Friedreich’s ataxia (FA), has joined the company as the co-chairman of its scientific advisory board. Professor…
The U.S. Food and Drug Administration’s (FDA) Office of Orphan Products Development recently granted orphan drug designation to Retrotope‘s stabilized fatty acid drug (RT001) for the treatment of Friedreich’s taxia (FA). The FDA designation follows a statement from the company that RT001 was well tolerated by patients with FA without…
Global Genes, a leading rare genetic disease advocacy organization, has just announced the winners of its inaugural RARE Patient Impact Grant Program, an initiative first launched at the 2015 Rare Tribute to Champions of Hope. Included in the awardees is the Friedreich’s ataxia leading advocacy organization, Friedreich’s Ataxia…
Agilis Biotherapeutics, LLC, and Waisman Biomanufacturing have signed an exclusive partnership agreement to develop and produce AGIL-FA, Agilis’ potential gene therapy for the treatment of Friedreich’s ataxia (FA). Agilis, a biotechnology company specializing in therapies for rare genetic diseases that affect the central nervous system (CNS), and Waisman, a nonprofit gene…
University of Texas (UT) Southwestern Medical Center researchers have identified RNA and DNA synthetic agents that increase frataxin levels and alleviate Friedreich’s ataxia (FA), suggesting that synthetic nucleic acids that target the specific disease-causing mutation can be lead compounds for FA therapy. The research paper, “Activating frataxin expression by repeat-targeted nucleic acids,”…
Retrotope announced the completion of a first dosing cohort, and the opening of patient enrollment for a highest dose cohort, in its ongoing, 28-day clinical study of orally dosed RT001 in Friedreich’s ataxia (FA) patients. The treatment to date has been well-tolerated, with no serious adverse effects or dose limiting toxicities, the pharmaceutical company reported. “We…
Researchers have demonstrated in the lab (in vitro) and in living subjects (in vivo), that gene transfer of brain-derived neurotrophic factor (BDNF) protected models of Friedreich’s ataxia from neuronal degenerative death, providing a proof of principle that neurotrophins might be a therapeutic option to prevent and treat neurodegeneration associated with Friedreich’s ataxia.
RaNA Therapeutics, a Cambridge, Massachusetts-based biotech company that develops next-generation RNA-targeted medicines and which currently has a lead program in Friedreich’s Ataxia, announced that its CEO Ronald Renaud will present a corporate overview on Feb. 8 at the 18th annual BIO CEO & Investor Conference in New York City. RaNA’s management will also…
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