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...
The UK government is making it easier for millions of people to participate in clinical trials and is boosting transparency around how studies are delivered across the National Health Service.
The European Medicines Agency expects the International Council for Harmonization’s new guideline will help address the critical lack of data on the use of medicinal products in pregnant and breastfeeding populations.
A differentiated profit-sharing model to accelerate the use of AI in drug development using clinical and patient data is being proposed in South Korea, but the idea faces multiple practical challenges
