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

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

CONNECT1 has enrolled two cohorts of boys and young men living with DMD amenable to exon 51 skipping and its endpoints include safety and tolerability, dystrophin production, exon skipping ...

PGN-EDO51, designed to skip exon 51 in the dystrophin transcript, could potentially restore the production of a functional dystrophin protein for approximately 13% of DMD patients. The FDA has ...

Multiple companies are digging in to add the next generation of exon skippers, gene therapies and medicines targeting more than skeletal muscle to their DMD pipelines, and for Michael Kelly, chief ...

The SALSA MC002 SMA Newborn Screen uses PCR amplification and melt curve analysis to determine the presence or absence of exon 7 in the SMN1 and SMN2 genes from a dried blood spot sample.

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