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 ...

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

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.

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

The rapid development of gene therapy options for treating neuromuscular diseases has created new therapeutic options but ...

The Phase I/II Deliver trial sought to establish the impact of DYNE-251 on several mobility-related endpoints, seeing an ...

Duchenne muscular dystrophy (DMD) is a fatal genetic disorder caused by mutations in the DMD gene. Here, the authors develop MyoAAV-UA, a compact utrophin activator, as a promising universal ...

Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, will present data from the Phase 1/2 INSPIRE DUCHENNE ...

An experimental gene therapy for Duchenne Muscular Dystrophy (DMD) has showed better-than-expected results in a three-patient trial, according to preliminary data. Company shares jumped 60 percent ...

New Mexico officials have announced the cause of death for actor Gene Hackman and his wife Betsy Arakawa, a week after they were found dead at their home in Santa Fe. Hackman died of heart disease ...

Gene was suffering from severe heart disease and advanced Alzheimer's disease, while Betsy suffered from hantavirus, which is spread through rodent droppings Gene Hackman's wife Betsy Arakawa ...