Patients’ and caregivers’ maximum acceptable risk of death for non‐curative gene therapy to treat Duchenne muscular dystrophy-Reference-Cited by-同舟云学术

Patients’ and caregivers’ maximum acceptable risk of death for non‐curative gene therapy to treat Duchenne muscular dystrophy

Published:2021-03-23 Issue: Volume: Page:
ISSN:2324-9269
Container-title:Molecular Genetics & Genomic Medicine
language:en
Short-container-title:Mol Genet Genomic Med

Author:

Peay Holly L.¹^ORCID,Fischer Ryan²,Mange Brennan³,Paquin Ryan S.¹,Smith Edward C.⁴,Sadosky Alesia⁵,Russo Leo⁵,Ricotti Valeria⁶,Rensch Colin²,Morris Carl⁶,Martin Amy Strong⁷,Ganot Annie⁶,Beaverson Katherine⁵,Mansfield Carol³

Affiliation:

1. RTI International Research Triangle Park NC USA

2. Parent Project Muscular Dystrophy Hackensack NJ USA

3. RTI Health Solutions Research Triangle Park NC USA

4. Duke University Durham NC USA

5. Pfizer, Inc New York NY USA

6. Solid Biosciences Cambridge MA USA

7. UCLA Los Angeles CA USA

Publisher

Wiley

Subject

Genetics (clinical),Genetics,Molecular Biology

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1002/mgg3.1664

Reference25 articles.

1. FDA Approves Eteplirsen for Duchenne Muscular Dystrophy: The Next Chapter in the Eteplirsen Saga

2. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management

3. Progress toward Gene Therapy for Duchenne Muscular Dystrophy

4. The control preferences scale;Degner L. F.;Canadian Journal of Nursing Research,1997

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