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 ...

Tevard Biosciences, Inc., a privately held biotechnology company pioneering tRNA-based therapies to cure a broad range of ...

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

Characterized by progressive muscle degeneration, this X-linked disorder results from mutations in the DMD gene, which encodes dystrophin, a protein essential for maintaining muscle cell integrity.

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 ...

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 ...

They report that in mice, HG302 restored dystrophin protein levels up to 70% and improved motor function. Safety evaluations showed no abnormal liver enzymes or kidney function in both the mice ...

This gene is responsible for producing the dystrophin protein, which plays an important role in keeping muscle cells intact. It acts like a shock absorber in muscle cells, helping to protect muscle ...