Sarepta Therapeutics’ Gene Therapy Limb-Girdle Muscular Dystrophy Type 2E Clinical Data has been Accepted for a Late-breaking Oral Presentation at the 2019 MDA Clinical and Scientific Conference
Title: Systemic Gene Transfer with AAVrh74.MHCK7.SGCB Increased β-sarcoglycan Expression in Patients with Limb Girdle Muscular Dystrophy Type 2E (Rodino-Klapac et al.)
In addition to the late-breaker, six posters highlighting data from Sarepta’s RNA and gene therapy programs for Duchenne muscular dystrophy (DMD) and LGMD 2D will be presented during the Networking & Poster Sessions in the Exhibit Hall on
The oral presentation and posters will be archived under the events and presentations section of the
About 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 5 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. For more information, please visit www.sarepta.com.
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Source: Sarepta Therapeutics, Inc.
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Sarepta Therapeutics 2019 J.P. Morgan Healthcare Conference Presentation