Download Attachments
Sarepta Therapeutics Sarepta is at the forefront of precision genetic medicine, having built an impressive and competitive position in Duchenne muscular dystrophy (DMD) and more recently in gene therapies for 6 Limb-girdle muscular dystrophy diseases (LGMD), Charcot-Marie-Tooth (CMT), MPS IIIA, Pompe and other CNS-related disorders, totaling over 20 therapies in various stages of development. The Company’s programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. Sarepta is fueled by an audacious but important mission: to profoundly improve and extend the lives of patients with rare genetic-based diseases.
The purpose of the Sarepta Therapeutics Opinion Paper is to provide a rational and feasible approach to monitoring patients with Duchenne muscular dystrophy (DMD) in a clinical setting to better assist providers and insurers in assessing response to treatment compared to the natural history of DMD. Patient groups with different genetic mutations vary in their expected rates of decline, so genotype-specific natural history should inform evaluations when available. To learn more, read the opinion paper on Outcome Measures for Patients With Duchenne Muscular Dystrophy.
**Please note: This paper represents the views of the author, not the Ohio Association of Health Plans (OAHP). The publication, distribution or posting of this paper by OAHP does not constitute a guarantee of any product or service by OAHP.