A survey of 86 German patient groups suggests pharma’s reputation is improving in the country that has been wary of pharma in the past, with Amgen and Janssen leading the way.
Making sure everyone with DMD gets access to treatments and the ‘essential’ interventions that can make life worth living is key.
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.
US FDA expert committee’s ‘yes’ could pave the way for a new generation of gene therapies for rare inherited eye disorders.
Benefitting from insights from focus groups with rare disease patients.