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Specialty
Massage Therapy
A leader in pediatric massage therapy, Nationwide Children’s is one of the only children’s hospitals to staff a department of full-time, licensed massage therapists.
News
Phase 1 Study Shows Encouraging Data for Gene Replacement Therapy for Spinal Muscular Atrophy Type 1
A one-time intravenous infusion of the high dose of gene therapy extended the survival of patients with spinal muscular atrophy type 1 (SMA1) in a Phase 1 clinical trial, according to a study published today in the New England Journal of Medicine. The study was conducted by Researchers from Nationwide Children’s Hospital in collaboration with AveXis, Inc. and The Ohio State University College of Medicine.
News
AveXis Receives U.S. FDA Breakthrough Therapy Designation for AVXS-101 Gene Replacement Therapy for Spinal Muscular Atrophy Type 1
AveXis, Inc., (Nasdaq:AVXS) a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced the U.S.
Specialty
Spinal Muscular Atrophy Clinic
The SMA Clinic is a joint program between Nationwide Children’s and The Ohio State University Wexner Medical Center. Our team provides comprehensive, multidisciplinary care for patients from across the United States.
Condition
Becker Muscular Dystrophy
Becker muscular dystrophy is the same basic disease as Duchenne muscular dystrophy, except it is less common and the symptoms are milder and slower to progress. It only affects males. Once symptoms begin, they follow a slower, but similar, course to that of Duchenne muscular dystrophy.
Article
July 2011
Dr. Carsten Bonnemann discusses collagen VI myopathies and a novel cause of ullrich congenital muscular dystrophy.
Article
March 2015
Dr. Jerry Mendell discusses phase 1/2a follistatin gene therapy trial for becker muscular dystrophy.
Myotonic Muscular Dystrophy
Myotonic muscular dystrophy, also known as MMD or Steinert’s disease, is the most common form of MD in adults.