Vamshi K. Rao, MD

Final thoughts from leading experts on the future of treating Duchenne muscular dystrophy and keeping standard of care in the forefront.

Recent approval of casimersen, its administration, efficacy, safety, and patient selection for treatment of Duchenne muscular dystrophy.

Viltolarsen phase II study data is discussed as well as a comparison of viltolarsen to golodirsen for treatment of Duchenne muscular dystrophy.

A review of exon skipping agents, their function, mechanism of action, and the different drugs used in clinical practice to treat Duchene muscular dystropy.

A discussion with parents on treatment options in Duchenne muscular dystrophy as well as the benefit of using steroids combined with other therapies.

Discussion on the importance of early treatment in patients with Duchenne muscular dystrophy.

Eric P. Hoffman, PhD, outlines the variables in measurement of dystrophin levels in patients with Duchenne muscular dystrophy and how they translate into clinical benefit.

Consideration of the measurement and standardization of dystrophin improvement to infer clinical benefit and the FDA distinction between exon skipping dystrophin and micro-dystrophin in Duchenne muscular dystrophy. 

Discussion of micro-dystrophin, its place in gene therapy, and relevance in the clinical setting in relation to Duchenne muscular dystrophy.

Vamshi K. Rao, MD, and Eric P. Hoffman, PhD, review Duchenne muscular dystrophy and the mutations involved.