Encuentra rápidamente lo que buscas

Journal of Neuromuscular Diseases
Journal of Neuromuscular Diseases
Volume 11, issue 5

Todas las revistas Índice de la revista

Lek, Angela | Atas, Evrim | Lin, Brian | Hesterlee, Sharon E. | Abbott, Jordan K. | Byrne, Barry J. | Bönnemann, Carsten G.

This meeting report summarizes the presentations and discussions held at the summit on Challenges in Gene Therapy hosted by the Muscular Dystrophy Association (MDA) in 2023. Topics covered include safety issues, mitigation strategies and practical considerations pertaining to the clinical translation of gene therapies for neuromuscular disease. The listing of actionable recommendations will assist in overall efforts in the field to achieve safe and efficacious translation of gene therapies for neuromuscular disease patients.