Improving study recruitment with patient-centric trial endpoints
Making sure everyone with DMD gets access to treatments and the ‘essential’ interventions that can make life worth living is key.
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.
Shares in Solid Biosciences plunged yesterday after the FDA placed a clinical hold on the trial of its gene therapy for Duchenne muscular dystrophy (DMD) following a serious adverse reactions in one patient.