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Molecular Genetics & Genomic Medicine
|
March 23, 2021
Patients' and caregivers' maximum acceptable risk of death for non-curative gene therapy to treat Duchenne muscular dystrophy
Holly L Peay, Ryan Fischer, Brennan Mange, et al.
Orphanet Journal of Rare Diseases
|
May 11, 2019
Priorities when deciding on participation in early-phase gene therapy trials for Duchenne muscular dystrophy: a best-worst scaling experiment in caregivers and adult patients
Ryan S Paquin, Ryan Fischer, Carol Mansfield, et al.
Neuromuscular Disorders : NMD
|
December 27, 2022
Clinical potential of microdystrophin as a surrogate endpoint
Jessica F Boehler, Kristy J Brown, Margaret Beatka, et al.
Journal of Neuromuscular Diseases
|
August 2, 2024
Clinician Perspectives of Gene Therapy as a Treatment Option for Duchenne Muscular Dystrophy
Heidi Cope, Ryan Fischer, Emma Heslop, et al.
European Journal of Medical Genetics
|
October 11, 2025
Re-evaluating acceptable risk of death from gene therapy: A threshold study among individuals with Duchenne muscular dystrophy and their caregivers in the US and UK
Holly Peay, Ryan Fischer, Megan McNiff, et al.
Page
of 7
Search research articles
Search
Showing results (61-70 of 65) with videos related to
Sort By:
Page
of 7
You have reached the last page of results.
This site can display upto 65 results.
Molecular Genetics & Genomic Medicine
|
March 23, 2021
Patients' and caregivers' maximum acceptable risk of death for non-curative gene therapy to treat Duchenne muscular dystrophy
Holly L Peay, Ryan Fischer, Brennan Mange, et al.
Orphanet Journal of Rare Diseases
|
May 11, 2019
Priorities when deciding on participation in early-phase gene therapy trials for Duchenne muscular dystrophy: a best-worst scaling experiment in caregivers and adult patients
Ryan S Paquin, Ryan Fischer, Carol Mansfield, et al.
Neuromuscular Disorders : NMD
|
December 27, 2022
Clinical potential of microdystrophin as a surrogate endpoint
Jessica F Boehler, Kristy J Brown, Margaret Beatka, et al.
Journal of Neuromuscular Diseases
|
August 2, 2024
Clinician Perspectives of Gene Therapy as a Treatment Option for Duchenne Muscular Dystrophy
Heidi Cope, Ryan Fischer, Emma Heslop, et al.
European Journal of Medical Genetics
|
October 11, 2025
Re-evaluating acceptable risk of death from gene therapy: A threshold study among individuals with Duchenne muscular dystrophy and their caregivers in the US and UK
Holly Peay, Ryan Fischer, Megan McNiff, et al.
Page
of 7