US biotech Sarepta's shares jumped by as much as 60% after a gene therapy produced dramatic results in three boys with the muscle wasting disease Duchenne muscular dystrophy (DMD).
Santhera is putting patient needs at the heart of its rare disease speciality business. Chief Executive Officer Thomas Meier, PhD, talks about the company’s mission – and how rationalising Europe’s drug review systems could help patients.
Progress in health and social care in European countries is helping many boys with Duchenne muscular dystrophy (DMD) to extend their lives beyond what was once dreamed possible, and to take control of their lives as they grow up and become men.