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New Promise, Looming Competition For DMD In China

70,000 Patients, No Approved Drugs

 
• By Brian Yang and Kosh Naran

With several new drugs progressing for the estimated 70,000 patients in China with DMD, a recent setback for Nippon Shinyaku's contender and progress for Chinese gene therapy developers are adding both uncertainty and likely competition in an untapped market with pressing medical need.

Muscular dystrophy wheelchair user
DMD patients in China sees promise but also uncertainties • Source: Shutterstock

In a recent discussion forum on China's WeChat social media platform, a group of patients whose sons are suffering from Duchenne muscular dystrophy (DMD) had good news to share - neurologists at one of the nation’s top-ranked medical facilities, the Beijing Union Hospital, could finally prescribe PTC Therapeutics, Inc.'s Emflaza (deflazacort).

Approved in the US in early 2017 as the first drug for the devasting, severe and progressive genetic muscle wasting...

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