Gene Therapy for Muscular Disorders
The ONLY digital meeting in this space!
Ellie Saunders
Duchenne, gene therapy, Muscular disorders, muscular dystrophy, Sarepta
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The ONLY digital meeting in this space!
Disease Spotlight/ DMD Main/ Views and analysis
Dominic Tyer
disease, DMD, DMD campaigners, DMD campaigning, Duchenne, Klair Bayley, Parent Project Muscular Dystrophy, Pat Furlong, Ricky Andreis, Santhera, Save Our Sons, Swedish Muscular Dystrophy Research Foundation
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Piotr Wnuk
DMD, Duchenne, Duchenne muscular dystrophy, Pfizer, rare disease
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Disease Spotlight/ DMD Main/ Patients/ Views and analysis
Linda Banks
DMD, Duchenne, Duchenne muscular dystrophy, patient group, patient organisation
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Market Access/ Patients/ Views & Analysis/ Views & Analysis/ Views and analysis
Linda Banks
DMD, Duchenne, Duchenne muscular dystrophy, LHON, patient centricity
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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.
Disease Spotlight/ DMD Main/ Patients/ Views and analysis
Linda Banks
DMD, Duchenne, Duchenne muscular dystrophy, patient centricity, patient perspective
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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.
Articles/ Disease Spotlight/ DMD Main/ Views and analysis
Linda Banks
DMD, Duchenne, Duchenne muscular dystrophy, patients, rare disease
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Andrew McConaghie
Bernie Sanders, Duchenne, Marathon Pharmaceuticals, Rare diseases
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Company says it will listen to concerns of patient advocates