Interim results from a small group of children in a Phase I/II trial are essentially in line with that of Elevidys, according ...

Gene therapies, exon-skipping drugs and next-gen steroids are reshaping Duchenne muscular dystrophy care, targeting broader ages and mutations. We are witnessing the fastest pivot in rare-disease ...

The Platform Technology Designation, which precedes the current FDA leadership, is designed to streamline the drug ...

RegenXBio has shared updated phase 1/2 data on its Duchenne muscular dystrophy (DMD) gene therapy, providing evidence that ...

The company is one of the first to receive a “platform technology designation,” which could speed the review of certain gene ...

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Phil Lambert, Ph.D., chief scientific officer of Satellos Bioscience Inc., explains the mechanism of action of the company's experimental Duchenne muscular dystrophy drug, SAT-3247, and the thinking ...

Dystrophin's isoforms are thought to contribute variably to neuropsychiatric disorders, behavioral disorders, and ...

Cardiac complications in Duchenne muscular dystrophy (DMD) are the result of cardiac muscle involvement that accompanies the ...

Intellia’s stock fell 23% after one patient in its Phase III MAGNITUDE trial developed severe liver toxicity that later ...

Advancements in gene therapy for Duchenne muscular dystrophy (DMD) have improved the outlook for some patients, but further ...

Intellia's news comes at a challenging time for gene therapies, as multiple serious adverse events and fatalities have been ...