Editor’s note: This is an automatically generated transcript, which has been slightly edited for clarity. Please notify [email protected] if there are concerns regarding accuracy of the transcription.
Editor’s note: This is an automatically generated transcript, which has been slightly edited for clarity. Please notify [email protected] if there are concerns regarding accuracy of the transcription.
Panelists discuss how the differential diagnosis for Friedreich's Ataxia involves distinguishing it from other ataxias, the process of definitive diagnosis through genetic testing, and the significant ...
The National Ataxia Foundation (NAF) is an organization devoted to raising awareness, supporting families, and funding research for Ataxia, a rare and progressive neurological condition that impairs ...
A University of Oklahoma researcher is the first to discover that the sequence of the genetic defect in the neuromuscular disease Friedreich's ataxia isn't always as uniform as previously thought, a ...
Larimar Therapeutics’ Friedreich’s ataxia program is free at last, with the FDA fully lifting a clinical hold that had remained in part on the program since 2021. The rare disease biotech can now ...
The US Food and Drug Administration (FDA) has rejected PTC Therapeutics’ new drug application (NDA) for vatiquinone in Friedreich's Ataxia. This follows the biotech’s decision to submit the ...
Biogen Inc. (NASDAQ:BIIB) is among the 11 Best Genomics Stocks to Buy According to Hedge Funds. It has begun dosing in the global Phase 3 BRAVE trial to test omaveloxolone (SKYCLARYS®) in children ...
Several months after forging a fresh path forward for its troubled Friedreich ataxia (FA) drug vatiquinone, PTC Therapeutics is back with some much-needed positive data. The candidate passed muster by ...
Some results have been hidden because they may be inaccessible to you
Show inaccessible results
Feedback