Sarepta Therapeutics presents positive picture at ICNMD 2022
Cambridge, USA-based gene therapy company Sarepta Therapeutics (Nasdaq: SRPT) has released positive data from multiple studies investigating its Duchenne muscular dystrophy (DMD) candidate SRP-9001.
Results to be presented this week at the 17th International Congress on Neuromuscular Diseases (ICNMD 2022) show positive results across several time points, including after one, two and four years of treatment.
The investigational gene transfer therapy is designed to deliver a micro-dystrophin-encoding gene to muscle tissue, enabling the body to produce this essential protein.
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