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Duchenne muscular dystrophy (DMD) is a rare hereditary disease that is associated with progressive muscle wasting. The ...

Results show improved functional outcomes for people with Duchenne muscular dystrophy who are ambulatory. Elevidys, which is ...

Utrophin increase in muscle cells after transcriptional adaptation normalises cell function in Duchenne muscular dystrophy ...

Cumberland Pharmaceuticals Inc. (Nasdaq: CPIX), a specialty pharmaceutical company with development efforts focused on new products for rare diseases, today announced positive top-line results from ...

Entrada has secured authorisation from the UK's MHRA to commence ELEVATE-44-201, to evaluate ENTR-601-44, aimed at treating DMD.

Duchenne muscular dystrophy (DMD) is a rare genetic disorder that is characterised by progressive muscle degeneration and weakness, and is caused by mutations in the dystrophin gene. Currently ...

Cumberland Pharmaceuticals Inc. (NASDAQ: CPIX) shares began Tuesday sharply higher. Cumberland, a specialty pharmaceutical company with development efforts focused on new products for rare diseases, ...

The gene therapy improved motor functions in children with Duchenne muscular dystrophy two years after treatment ...

Around 13% of DMD patients respond to exon 51 skipping, but PepGen reckons other drugs in its pipeline will address mutations that are central to around 35% of all cases of the disease.