Duchenne Muscular Dystrophy: Pipeline And Partnering Snapshot

A full pipeline of symptomatic and disease-modifying treatments for Duchenne will transform a long dormant orphan market into a hotly contested commercial prize. But some issues, foremost the scarcity of trial patients, threaten to stall progress in the field.

Duchenne muscular dystrophy is the latest rare disease market to attract the attention of investors and big pharma. It may also be the launching pad for the next turbo-charged small-cap developer, as Sarepta Therapeutics Inc. demonstrated recently with its release of promising Phase IIB results.

Numerous assets addressing a variety of biological targets – early and late stage, partnered and unpartnered – are advancing through...

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