Annie Ganot

Priorities when deciding on participation in early-phase gene therapy trials for Duchenne muscular dystrophy: a best–worst scaling experiment in caregivers and adult patients

V. Ricotti, C. Mansfield, Edward C Smith, 2019, Orphanet Journal of Rare Diseases.

Clinical potential of microdystrophin as a surrogate endpoint

J. Chamberlain, M. Lawlor, M. Beatka, 2022, Neuromuscular Disorders.

Microdystrophin Expression as a Surrogate Endpoint for Duchenne Muscular Dystrophy Clinical Trials

O. Danos, D. Duan, J. Chamberlain, 2023, Human gene therapy.

Patients’ and caregivers’ maximum acceptable risk of death for non‐curative gene therapy to treat Duchenne muscular dystrophy

V. Ricotti, C. Mansfield, A. Sadosky, 2021, Molecular Genetics & Genomic Medicine.