Lessons learned about gene therapy for neuromuscular disorders

In a panel discussion held at WMS 2020, experts in the field of gene therapy for neuromuscular disorders (NMDs) discussed dosing and safety issues for novel gene-based therapies.

The key points of the discussion included:

    Dr Ana Buj Bello (Genethon, Evry, France) reviewed the dosing of different gene-based therapies for Duchenne muscular dystrophy (DMD), spinal muscle atrophy (SMA), and myotubular myopathy (MTM). Clinical data suggest that higher doses result in higher protein expression in target tissue. “What is the right dose for patients?” Dr Bello asked. “This is an important question that needs to be analyzed carefully with preclinical and clinical trials.”