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Clinician Perspectives of Gene Therapy as a Treatment Option for Duchenne Muscular Dystrophy.
Cope, Heidi; Fischer, Ryan; Heslop, Emma; McNiff, Megan; Johnson, Alexandra; Camino, Eric; Denger, Brian; Armstrong, Niki; Thakrar, Sejal; Bateman-House, Alison; Beaverson, Katherine L; Woollacott, Ione O C; Phillips, Dawn; Fernandez, Vivian; Ganot, Annie; Donisa-Dreghici, Roxana; Mansfield, Carol; Peay, Holly.
Afiliación
  • Cope H; RTI Health Solutions, Research Triangle Park, NC, USA.
  • Fischer R; Parent Project Muscular Dystrophy, Washington, D.C., USA.
  • Heslop E; John Walton Muscular Dystrophy Research Centre, Newcastle University, Newcastle Upon Tyne, UK.
  • McNiff M; John Walton Muscular Dystrophy Research Centre, Newcastle University, Newcastle Upon Tyne, UK.
  • Johnson A; Duchenne UK, London, UK.
  • Camino E; Parent Project Muscular Dystrophy, Washington, D.C., USA.
  • Denger B; Parent Project Muscular Dystrophy, Washington, D.C., USA.
  • Armstrong N; Parent Project Muscular Dystrophy, Washington, D.C., USA.
  • Thakrar S; Duchenne UK, London, UK.
  • Bateman-House A; Smile with Shiv, UK.
  • Beaverson KL; Department of Population Health, Division of Medical Ethics, NYU Grossman School of Medicine, New York, NY, USA.
  • Woollacott IOC; Pfizer, Inc. New York, NY, USA.
  • Phillips D; Pfizer Ltd., Tadworth, Surrey, UK.
  • Fernandez V; Regenxbio Inc., Rockville, MD, USA.
  • Ganot A; Regenxbio Inc., Rockville, MD, USA.
  • Donisa-Dreghici R; Solid Biosciences Inc., Charlestown, MA, USA.
  • Mansfield C; Solid Biosciences Inc., Charlestown, MA, USA.
  • Peay H; RTI Health Solutions, Research Triangle Park, NC, USA.
J Neuromuscul Dis ; 11(5): 1085-1093, 2024.
Article en En | MEDLINE | ID: mdl-39093077
ABSTRACT

Background:

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.

Objective:

This study aimed to understand the perspectives of clinicians about gene therapy for DMD, which has not previously been studied.

Methods:

We conducted interviews with specialist clinicians treating patients with DMD in the United States (n = 8) and United Kingdom (n = 8). Interviews were completed in 2022, before any approved gene therapies, to gain insight into barriers and facilitators to implementing gene therapy and educational needs of clinicians.

Results:

Most respondents expressed cautious optimism about gene therapy. Responses varied regarding potential benefits with most expecting delayed progression and duration of benefit (1 year to lifelong). Concern about anticipated risks also varied; types of anticipated risks included immunological reactions, liver toxicity, and cardiac or renal dysfunction. Clinicians generally, but not uniformly, understood that gene therapy for DMD would not be curative. Most reported needing demonstrable clinical benefit to justify treatment-related risks.

Conclusions:

Our data demonstrate variability in knowledge and attitudes about gene therapy among clinicians who follow patients with DMD. As our knowledge base about DMD gene therapy grows, clinician education is vital to ensuring that accurate information is communicated to patients and families.
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Terapia Genética / Distrofia Muscular de Duchenne Límite: Female / Humans / Male País/Región como asunto: America do norte / Europa Idioma: En Revista: J Neuromuscul Dis Año: 2024 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Países Bajos

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Terapia Genética / Distrofia Muscular de Duchenne Límite: Female / Humans / Male País/Región como asunto: America do norte / Europa Idioma: En Revista: J Neuromuscul Dis Año: 2024 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Países Bajos