Scientists are seeking new grant opportunities, founding their own for-profit ventures, and exploring other approaches to ...

New data demonstrated the benefits of Elevidys, a gene therapy for multiple Duchenne muscular dystrophy, in different age ...

Gene therapy could be the newest breakthrough for Duchenne muscular dystrophy. While it may not be a cure, it can give independence to boys diagnosed with DMD.

The Janus Henderson Global Life Sciences Fund returned 0.99% and the MSCI World Health Care IndexSM returned 5.10% for Q1 ...

Canadians are walking, running, and rolling in support of the 30th annual Walk to Defeat Duchenne, the country's only ...

Multiple first-in-human trials showed functional and biomarker improvements in early data reported at ASGCT's annual meeting.

In cohort 6, subjects aged two years old at the time of treatment showed a mean expression of 93.87% of normal dystrophin ...

On first glance, Dr. Vinay Prasad’s medical credentials sound impressive. But look closer and you’ll find a narcissistic ...

The gravity of these decisions calls for a degree of humility on the part of an FDA leader. And that’s the rub with Vinay ...

Sarepta Therapeutics is meeting with the FDA in June to discuss expanding the use of its Duchenne muscular dystrophy gene ...

Since Elevidys' accelerated approval in 2023, experts have been clamoring for more data, particularly in older and ...

Muscle weakness has the biggest impact on patients’ ability to walk, but other factors matter too, researchers found.