Future of Treating DMD
Final thoughts from leading experts on the future of treating Duchenne muscular dystrophy and keeping standard of care in the forefront.
Casimersen in the Clinical Setting
Recent approval of casimersen, its administration, efficacy, safety, and patient selection for treatment of Duchenne muscular dystrophy.
Viltolarsen Phase II Study
Viltolarsen phase II study data is discussed as well as a comparison of viltolarsen to golodirsen for treatment of Duchenne muscular dystrophy.
Exon Skipping Agents Used in DMD
A review of exon skipping agents, their function, mechanism of action, and the different drugs used in clinical practice to treat Duchene muscular dystropy.
Use of Steroids in Combination With Other Treatments in DMD
A discussion with parents on treatment options in Duchenne muscular dystrophy as well as the benefit of using steroids combined with other therapies.
Early Treatment of DMD
Discussion on the importance of early treatment in patients with Duchenne muscular dystrophy.
Varying Dystrophin Levels in DMD
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.
Measuring Dystrophin in DMD
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.
Micro-Dystrophin in Gene Therapy
Discussion of micro-dystrophin, its place in gene therapy, and relevance in the clinical setting in relation to Duchenne muscular dystrophy.
Overview of Duchenne Muscular Dystrophy (DMD)
Vamshi K. Rao, MD, and Eric P. Hoffman, PhD, review Duchenne muscular dystrophy and the mutations involved.
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