PREDNISOLONE, METHYLPREDNISOLONE, Rapamune 1 mg/mL oral solution, rAAV8-hSGCG clinical trials

A Phase I/II, open-label, dose escalation study to evaluate the safety of 2 doses of intravenous ATA-200, an adeno-associated viral vector carrying the human gamma-sarcoglycan gene, in patients with gamma-sarcoglycanopathy (limb-girdle muscular dystrophy LGMDR5, formerly LGMD2C).

This study is testing a new gene therapy for a rare muscle condition called limb-girdle muscular dystrophy type R5 (LGMD-R5). It aims to see if the treatment is safe, what dose works best, and if it helps improve muscle function.