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PTC Readies China Plan Amid US FDA Translarna Setback

 
• By Brian Yang

PTC is engaging physicians and Duchenne muscular dystrophy patients in China to access a largely untapped market but regulatory and reimbursement-related uncertainties could pose challenges.

Hundreds of people – parents of young boys and young men on wheelchairs – recently listened intently to talk after talk, 16 in all in a single day, at a packed hotel ballroom in Beijing. They were mostly parents of Duchenne muscular dystrophy (DMD) patients.

Many of them had travelled hundreds of miles from across the country to attend the 5th DMD Patients and Physician...

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