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SLDB Stock Up as FDA Clears IND for SGT-212 in Friedreich's Ataxia
Per the company, SGT-212 is the only full-length frataxin replacement gene therapy candidate that targets the cardiac manifestations of FA. SGT-212 is being developed to treat the neurologic and ...
Nasdaq1mon
Solid Biosciences Receives FDA Fast Track Designation for SGT-212, a Dual Route Gene Therapy for Friedreich’s Ataxia
This makes SGT-212 the only dual route gene transfer therapy for FA in development, using both intradentate nucleus and intravenous infusions to deliver the full-length frataxin gene. The therapy ...
Monthly Prescribing Reference24d
Vatiquinone Gets Priority Review for Friedreich Ataxia
FA is a physically debilitating, life-shortening, neuromuscular disorder caused by a defect in the frataxin gene; this defect causes decreased production of frataxin, a protein necessary for cell ...