Anne M. Connolly

Anne M. Connolly's work centers on clinical trials and long-term outcomes research for pediatric neuromuscular conditions, especially Duchenne muscular dystrophy and spinal muscular atrophy. Her publications frequently report results from multicenter drug and gene therapy trials, including safety, efficacy, and natural history studies of treatments such as gene therapies, antibody-based drugs, and exon-skipping therapies. This research is largely translational, involving collaboration across many clinical sites to evaluate new treatments in patients.

Publication output has fluctuated over the past decade with a peak around 2019-2021, followed by a decline and a lower but steady pace of publishing in recent years.

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