Jerry R. Mendell

This researcher focuses on gene therapy for spinal muscular atrophy (SMA), a genetic disease that weakens muscles and motor control, particularly in infants and children. Much of the work centers on onasemnogene abeparvovec, a gene therapy delivered via a viral vector to replace a missing or faulty gene (SMN1), and includes clinical trial results, long-term safety follow-up, and related muscular disorders like dysferlinopathy (a muscle-wasting condition). The research is largely clinical and translational, based on multi-site trials rather than laboratory-only studies.

Publication output has fluctuated over the past decade, peaking around 2019 and 2021, but has notably declined in the most recent two to three years.

Generated by claude-sonnet-5 from public bibliographic data · Jul 20, 2026