Decker was given the 2025 MDA Legacy Award, recognizing his efforts to advance gene therapy, scientific research, and the ...

Shares of Sarepta Therapeutics SRPT plunged nearly 24% last week after the company reported the death of a patient following ...

Research Triangle Park, N.C., March 07, 2025 (GLOBE NEWSWIRE) -- Data Safety Monitoring Board (DSMB) recommendation to initiate second cohort follows review of Phase 1/Phase 2 LION-CS101 trial ...

2021;6(11):887-896. [2] AskBio Announces First Patient Dosed in Phase 1 / Phase 2 Trial of AB-1003 Gene Therapy for Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9). Available at ...

A second higher dose will now be tested as Sarepta prepares for additional studies of SRP-9003 as well as gene therapies for other forms of LGMD, said the company’s chief executive Doug Ingram.

today announced the advancement of the Phase 1/Phase 2 LION-CS101 clinical trial of investigational gene therapy AB-1003 in patients with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9) with ...

Sarepta’s gene therapy pipeline extends beyond DMD, with a focus on limb-girdle muscular dystrophies (LGMD). The company’s LGMD gene therapy portfolio targets over 70% of LGMD patients, presenting ...