Sarepta is pressing forward with a bold plan to file with the FDA for accelerated approval of its gene therapy SRP-9001 for Duchenne muscular dystrophy (DMD) in the next few months, with a
New data on Sarepta's gene therapy for Duchenne muscular dystrophy (DMD) has gone a long way towards building confidence in its efficacy – but have introduced a concern about its safety.
Shares in Sarepta came under pressure after the biotech said it had temporarily halted a clinical trial of its new-generation Duchenne muscular dystrophy candidate, after a patient had a se
Just days after dosing its first patient in a Duchenne muscular dystrophy trial, US biotech PepGen has pushed the button on an initial public offering (IPO).
The development of gene therapies for Duchenne muscular dystrophy has proved to be challenging, but one of the key players – Sarepta – thinks it may have the clinical data needed to file fo
Pfizer has called a halt to a clinical trial of its gene therapy for Duchenne muscular dystrophy as it investigates the unexpected death of a young male patient.
A pharma company’s core mission is to improve patient outcomes. This hinges on effectively influencing HCP clinical behaviour and driving disease education.