Looking Forward to New Therapies: A Personal Perspective on the Translational Landscape for Muscular Dystrophies

Bushby K


Journal of Neuromuscular Diseases, July 2015
DOI: 10.3233/JND-150096

Abstract

Every patient hopes for a cure. Medicine across the centuries has expanded the numbers of diseases for which this is a realistic prospect, including many infections, surgically treatable conditions and now many cancers. For many other chronic conditions management with disease altering therapy is possible but not curative. The vast majority of these advances in medical care have been based on knowledge of the underlying cause of the disease, rendering them amenable to the development of specific therapy. With so many diseases now amenable to treatment, the diagnosis of an “incurable” condition is arguably more difficult to bear than ever before.

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