Muscular Dystrophy

Researchers are investigating the safety and effectiveness of a new gene therapy product that replaces the defective β-sarcoglycan gene.

While endocrinologists are not always part of a core DMD treatment team, their expertise is necessary, especially in handling the effects of long-term glucocorticoid therapy.

The model aims to provide the basis for many future studies to bring therapeutic options to patients with FSHD.

Louise Rodino-Klapac, PhD, principal investigator in the Center for Gene Therapy, shares an update on the advancement of LGMD2E gene therapy to clinical studies.