John F. Brandsema

Neuromusculoskeletal Medicine

3501 Civic Center Blvd
Philadelphia, PA 19104

Last Updated: 09/26/2026

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Found 35 publications

Cardiotoxic Effects and Microdystrophin Expression after Gene Therapy for DMD.

Cardiotoxic Effects and Microdystrophin Expression after Gene Therapy for DMD.

Journal: The New England journal of medicine

Published: July 22, 2026

Learnings from Patient Mortality after Delandistrogene Moxeparvovec Administration: A Report of Two Cases and Expert Committee Considerations for Future Mitigation and Management.

Learnings from Patient Mortality after Delandistrogene Moxeparvovec Administration: A Report of Two Cases and Expert Committee Considerations for Future Mitigation and Management.

Journal: Human gene therapy

Published: February 13, 2026

Differences in swallowing efficacy of disease modifying treatment between infants receiving pre-symptomatic and symptomatic administration.

Differences in swallowing efficacy of disease modifying treatment between infants receiving pre-symptomatic and symptomatic administration.

Journal: Orphanet journal of rare diseases

Published: August 21, 2025

Consensus recommendations and considerations for the delivery and monitoring of gene therapy in patients with Duchenne muscular dystrophy.

Consensus recommendations and considerations for the delivery and monitoring of gene therapy in patients with Duchenne muscular dystrophy.

Journal: Neuromuscular disorders : NMD

Published: June 17, 2025

Treatment Patterns and Disease Burden of Juvenile Myasthenia Gravis in the United States: A Cohort Study Using Health Care Claims Databases.

Treatment Patterns and Disease Burden of Juvenile Myasthenia Gravis in the United States: A Cohort Study Using Health Care Claims Databases.

Journal: Neurology

Published: June 09, 2025

Real-world outcomes of delandistrogene moxeparvovec gene therapy: Motor outcomes and emerging safety concerns.

Real-world outcomes of delandistrogene moxeparvovec gene therapy: Motor outcomes and emerging safety concerns.

Journal: Molecular therapy : the journal of the American Society of Gene Therapy

Published: May 12, 2025

Open-label phase IV trial evaluating nusinersen after onasemnogene abeparvovec in children with spinal muscular atrophy.

Open-label phase IV trial evaluating nusinersen after onasemnogene abeparvovec in children with spinal muscular atrophy.

Journal: The Journal of clinical investigation

Published: March 28, 2025

Pearls & Oy-sters: Severe Myotonic Crisis Resembling Malignant Hyperthermia.

Pearls & Oy-sters: Severe Myotonic Crisis Resembling Malignant Hyperthermia.

Journal: Neurology

Published: March 24, 2025

Long-Term Evaluation of Givinostat in Duchenne Muscular Dystrophy, and Natural History Comparisons.

Long-Term Evaluation of Givinostat in Duchenne Muscular Dystrophy, and Natural History Comparisons.

Journal: Annals of clinical and translational neurology

Published: February 27, 2025

Characterization of swallowing biomechanics and function in untreated infants with spinal muscular atrophy: A natural history dataset.

Characterization of swallowing biomechanics and function in untreated infants with spinal muscular atrophy: A natural history dataset.

Journal: Journal of neuromuscular diseases

Published: February 20, 2025

Outcomes of early-treated infants with spinal muscular atrophy: A multicenter, retrospective cohort study.

Outcomes of early-treated infants with spinal muscular atrophy: A multicenter, retrospective cohort study.

Journal: Muscle & nerve

Published: March 21, 2024

Subspecialty Health Care Utilization in Pediatric Patients With Muscular Dystrophy in the United States.

Subspecialty Health Care Utilization in Pediatric Patients With Muscular Dystrophy in the United States.

Journal: Neurology. Clinical practice

Published: November 14, 2023

Showing 1-12 of 35

Last Updated: 09/26/2026