News

Biogen Disappointed by INESSS Recommendation on SKYCLARYS™ and its Impact on Quebec Patients with Friedreich Ataxia
Biogen Canada Inc. is disappointed by INESSS's recommendation against listing SKYCLARYS™ (omaveloxolone), despite its therapeutic potential for individuals living with Friedreich ataxia (FA) — a ...
Design Therapeutics Announces Start of Friedreich Ataxia Patient Dosing Ex-U.S. in its RESTORE-FA Phase 1/2 Multiple-Ascending Dose Trial of DT-216P2
Initial Data from the Ongoing, Blinded Phase 1 Single-Ascending Dose Trial Has Demonstrated Favorable Safety and Pharmacokinetics IND Application Submitted to Expand RESTORE-FA Trial to U.S. Sites; ...
Gene editing disrupts Huntington's mutation in mice
Broad Institute researchers have developed a way to edit the genetic sequences at the root of Huntington's disease and ...
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A Gene Editing Strategy to Repair Huntington's Defects
Researchers have developed a gene editing strategy that can repair the genetic problems that lead to Huntington's disease and Friedreich's ataxia. | Genetics And Genomics ...
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Cardiac Complications of Duchenne Muscular Dystrophy
Cardiac complications in Duchenne muscular dystrophy (DMD) are the result of cardiac muscle involvement that accompanies the ...
Oklahoma teenager uses love of music to find a cure for rare neurological disease
An Edmond family is on a mission to find a cure. For a decade, Michael Gehr has been battling Friedreich's ataxia, a rare ...
Billionaire’s family backs Friedreich’s ataxia research fundraiser for friend Flynn Mitchell
Billionaire Graeme Hart's granddaughter Jemima Hawkesby and her friend Flynn Mitchell graduating. She is fundraising to help ...