Susan E. Matesanz

Neurology

3400 Civic Center Boulevard
South Pavilion, 2nd Floor
Philadelphia, PA 19104

Last Updated: 09/26/2026

Save publications for later

Sign Up

Not sure about your diagnosis?

Check Your Symptoms

Found 22 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

The phenotypic spectrum and genetic determinants of severe spinal muscular atrophy in individuals with a single SMN2 copy: an international retrospective observational study.

The phenotypic spectrum and genetic determinants of severe spinal muscular atrophy in individuals with a single SMN2 copy: an international retrospective observational study.

Journal: EClinicalMedicine

Published: December 17, 2025

Onasemnogene abeparvovec gene therapy for treatment of patients with spinal muscular atrophy: Updated real-world practical considerations.

Onasemnogene abeparvovec gene therapy for treatment of patients with spinal muscular atrophy: Updated real-world practical considerations.

Journal: Journal of neuromuscular diseases

Published: November 10, 2025

Real-world evidence on nusinersen treatment of persons with SMA: a focused review.

Real-world evidence on nusinersen treatment of persons with SMA: a focused review.

Journal: Journal of neuromuscular diseases

Published: October 25, 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

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

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

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

Journal: Neurology

Published: March 24, 2025

Current clinical applications of AAV-mediated gene therapy.

Current clinical applications of AAV-mediated gene therapy.

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

Published: January 17, 2025

Motor function testing rates and outcomes in Duchenne muscular dystrophy with comorbid autism and attention-deficit/hyperactivity disorder.

Motor function testing rates and outcomes in Duchenne muscular dystrophy with comorbid autism and attention-deficit/hyperactivity disorder.

Journal: Neuromuscular disorders : NMD

Published: October 17, 2024

Equitable Access of Delandistrogene Moxeparvovec for Patients With Duchenne Muscular Dystrophy: A Call for Discussion.

Equitable Access of Delandistrogene Moxeparvovec for Patients With Duchenne Muscular Dystrophy: A Call for Discussion.

Journal: Pediatric neurology

Published: August 04, 2024

Access to novel therapies for Duchenne muscular dystrophy-Insights from expert treating physicians.

Access to novel therapies for Duchenne muscular dystrophy-Insights from expert treating physicians.

Journal: Annals of the Child Neurology Society

Published: May 08, 2024

Showing 1-12 of 22

Last Updated: 09/26/2026