Last Updated: 09/26/2026
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Found 38 publications
Exosome-Mediated Benefits of Cell Therapy in Mouse and Human Models of Duchenne Muscular Dystrophy.
Journal: Stem cell reports
Published: March 14, 2026
Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial.
Journal: The Lancet. Neurology
Published: February 03, 2023
Absence of full-length dystrophin impairs normal maturation and contraction of cardiomyocytes derived from human-induced pluripotent stem cells.
Journal: Cardiovascular research
Published: February 11, 2019
AAV-Mediated Gene Transfer Restores a Normal Muscle Transcriptome in a Canine Model of X-Linked Myotubular Myopathy.
Journal: Molecular therapy : the journal of the American Society of Gene Therapy
Published: January 12, 2019
Exosome-Mediated Benefits of Cell Therapy in Mouse and Human Models of Duchenne Muscular Dystrophy.
Journal: Stem cell reports
Published: December 14, 2017
Cardiac involvement in female carriers of Duchenne or Becker muscular dystrophy.
Journal: Muscle & nerve
Published: April 11, 2017
Long-term effects of systemic gene therapy in a canine model of myotubular myopathy.
Journal: Muscle & nerve
Published: March 21, 2017
Systemic AAV8-Mediated Gene Therapy Drives Whole-Body Correction of Myotubular Myopathy in Dogs.
Journal: Molecular therapy : the journal of the American Society of Gene Therapy
Published: September 26, 2016
The utility of genetic testing in neuromuscular disease: A consensus statement from the AANEM on the clinical utility of genetic testing in diagnosis of neuromuscular disease.
Journal: Muscle & nerve
Published: August 22, 2016
Skeletal Muscle Pathology in X-Linked Myotubular Myopathy: Review With Cross-Species Comparisons.
Journal: Journal of neuropathology and experimental neurology
Published: January 30, 2016
Last Updated: 09/26/2026