To help neurologists, clinicians and families understand the current evidence for a new gene therapy for Duchenne muscular ...

Scientists present current evidence for a new gene therapy for Duchenne muscular dystrophy called delandistrogene moxeparvovec.

In this case the FDA said Sarepta’s data showing an increase in production of dystrophin – the protein that preserves muscle that is lacking in DMD patients – is likely to predict a clinical ...

This therapeutic approach aims to promote exon skipping in the nucleus, potentially halting or reversing the progression of DMD, a rare genetic condition characterised by mutations in the gene that ...

Scientists have previously completed preclinical studies in DMD mice and monkeys. They report that in mice, HG302 restored dystrophin protein levels up to 70% and improved motor function. Safety ...

Ezutromid was designed to treat DMD by boosting utrophin levels to compensate for the faulty dystrophin protein that is the root cause of the very rare muscle-wasting disease. Summit reported ...

To help neurologists, clinicians and families understand the current evidence for a new gene therapy for Duchenne muscular dystrophy ...

Duchenne muscular dystrophy (DMD) in India faces challenges in diagnosis, treatment, and support, highlighting the urgent ...