Gene Therapy for Muscular Disorders
Duchenne, gene therapy, Muscular disorders, muscular dystrophy, Sarepta
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The ONLY digital meeting in this space!
Duchenne
DMD campaigners: Advocating for change
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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Pfizer axes development of Duchenne muscular dystrophy antibody
DMD, Duchenne, Duchenne muscular dystrophy, Pfizer, rare disease
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Living with DMD: Inspiring each other’s futures
DMD, Duchenne, Duchenne muscular dystrophy, patient group, patient organisation
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Accelerating access for rare disease patients – the need to harmonise Europe’s regulation
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.
Can people with Duchenne be in charge of their own lives?
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.
Duchenne’s disease: A New Hope
DMD, Duchenne, Duchenne muscular dystrophy, patients, rare disease
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Marathon halts launch of Duchenne drug after price outcry
Bernie Sanders, Duchenne, Marathon Pharmaceuticals, Rare diseases
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Company says it will listen to concerns of patient advocates
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