Roles
- Professor of Clinical Neurology
- Director, Muscular Dystrophy Association Care Center
- Director, Charcot-Marie-Tooth Center of Excellence
- Principal Investigator - CMT Stem Cell Lab
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Biography
Clinical Neurologist and Neurophysiologist with special training and interest in neuromuscular conditions (particularly Inherited Peripheral Neuropathies). Senior Translational Scientist specialized in Cellular Reprogramming and Differentiation for disease modeling, with experience in both academic and biotechnology industry settings -
Education & Training
Education
Post Graduate Training
Licensures and Certifications
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Honors & Awards
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Teaching Interests
Charcot-Marie-Tooth disease
Neuromuscular Medicine
Neurogenetics
Neurology -
Research Interests
Genetic neuromuscular disorders
Genetic neuropathies (Charcot-Marie-Tooth disease)
Axonal degeneration
Drug discovery
Cellular reprogramming and differentiation -
Publications
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Clinical Trials
Viewing 0 of 2 clinical trials.
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A Multi-center, Randomized, Double-blind, Placebo Controlled Phase III Study to Assess the Efficacy, Safety, and Tolerability of PXT3003 in Charcot-Marie-Tooth Type 1A (CMT1A)
- Principal Investigator: Mario Saporta
- Study Phase: Phase 3
- Enrollment Status: Closed
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This is a clinical trial to investigate the efficacy of AT-007, an aldose reductase inhibitor, in the treatment of SORD-related neuropathy.
- Principal Investigator: Mario Saporta
- Study Phase: Phase II/III
- Enrollment Status: Closed