The dynamics of the Friedreich's ataxia market are anticipated to change as companies across the globe are thoroughly working toward the development of new drug therapy options to treat this disease.

today announced that the first Friedreich ataxia (FA) patient has been dosed via intravenous (IV) infusion in its RESTORE-FA (Reactivating Expression Suppressed Through Overcoming Repeat Expansion ...

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 Provided by GlobeNewswire Jun 4, 2025, 11:00:00 AM ...

June 04, 2025 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company developing treatments for serious degenerative genetic diseases, today announced that ...

Their results are described in a Nature Genetics paper published this week titled “Base editing of trinucleotide repeats that cause Huntington’s disease and Friedreich’s ataxia reduces ...

She is fundraising to help find a cure for Mitchell who has Friedreich's Ataxia. Photo / Supplied. Flynn Mitchell said one the lowest moments of his life was trying to get home safely in his ...

The expansion will add a new interventional radiology suite, MRI suite, procedure rooms, and patient recovery beds. The project will relocate the existing helipad and aims to address increased ...

The authors have constructed a brief timeline of major clinical research related to hereditary spastic paraplegia (HSP). This timeline summarizes the evolution of HSP research, from the first clinical ...

Matt Lafleur, who was told he had Friedreich ataxia as a child, is now on a mission of hope and advocacy for others. This story contains Matt Lafleur’s personal experience with SKYCLARYS ...

The Food and Drug Administration (FDA) has accepted for Priority Review the New Drug Application (NDA) for vatiquinone for the treatment of children and adults living with Friedreich ataxia (FA).

What Is Friedreich’s Ataxia? Friedreich’s ataxia (FA) is a rare condition. It causes damage to your nervous system that gets worse over time. As a result, you may have abnormal movements ...