Why didn’t analysts share the same fear that gripped investors about Sarepta stock? One reason is the known risk of acute liver injury associated with adeno-associated virus vector (AAV)-based gene ...

Things are going from bad to worse for Sarepta Therapeutics (NASDAQ: SRPT), a biotech focusing on gene therapies. The company ...

CRISPR technique can target single genes essential for cancer cell survival, toppling the whole structure, says nanomedicine ...

An 8-year-old northern Minnesota boy is one of the first in the country to receive a specific type of gene therapy treatment.

New Gene Therapy Approach Shows Promise for Duchenne Muscular Dystrophy July 24, 2024 — Researchers have made a significant breakthrough in developing a new gene therapy approach that restores ...

Study results support the role of functional dystrophin and suggest that delandistrogene moxeparvovec stabilizes or slows ...

The Company is currently analyzing the case and will update the prescribing information for Elevidys to note this development.

Sarepta stock may recover sharply as additional information and data reveal that the adverse event reported by the company is ...

After Sarepta reported the death of a patient who had recently taken the gene therapy Elevidys, patient advocacy group Parent ...

The interim data found one child in the Duchenne trial produced more than double the amount of the protein needed to maintain ...

Investigators from Insmed Inc. have presented new preclinical data on the efficacy of their adenoviral vector (AAV9)-based gene therapy INS-1201 for the treatment of Duchenne muscular dystrophy (DMD).