Clinician Perspectives of Gene Therapy as a Treatment Option for Duchenne Muscular Dystrophy

Heidi Cope; Ryan Fischer; Emma Heslop; M. McNiff; Alexandra Johnson; Eric Camino; Brian Denger; Niki Armstrong; Sejal Thakrar; Alison Bateman-House; Katherine Beaverson; Ione Woollacott; Dawn Phillips; Vivian Fernandez; Annie Ganot; Roxana Donisa-Dreghici; Carol Mansfield; Holly L. Peay

doi:10.3233/jnd-240033

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Abstract

Duchenne muscular dystrophy (DMD) is a progressive, life-limiting, neuromuscular disorder. Clinicians play an important role in informing families about therapy options, including approved gene therapies and clinical trials of unapproved therapies.

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Cope et al. (2024) studied this question.

synapsesocial.com/papers/68e5e6f9b6db64358757bd2b https://doi.org/10.3233/jnd-240033

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Clinician Perspectives of Gene Therapy as a Treatment Option for Duchenne Muscular Dystrophy

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Abstract

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Clinician Perspectives of Gene Therapy as a Treatment Option for Duchenne Muscular Dystrophy

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