Gene Therapy for Muscular Disorders
The ONLY digital meeting in this space!
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Gene Therapy for Muscular Disorders
https://pharmaphorum.com/events/gene-therapy-for-muscular-disorders/
Duchenne
Accelerating access for rare disease patients – the need to harmonise Europe’s regulation
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.
Can people with Duchenne be in charge of their own lives?
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.
Marathon halts launch of Duchenne drug after price outcry
Company says it will listen to concerns of patient advocates
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