Scientists and clinicians at Nationwide Children’s Hospital have launched a monthly podcast entitled, “This Month in Muscular Dystrophy,” featuring internationally known scientists discussing the latest research in muscular dystrophy and other neuromuscular disorders. The programs are posted each month at http://www.NationwideChildrens.org/muscular-dystrophy-podcast. The podcasts are geared toward patients, their families and primary care physicians who take care of patients with neuromuscular diseases.
Hosted by Kevin Flanigan, MD, an attending physician in Neurology at Nationwide Children’s Hospital and a principal investigator in the Center for Gene Therapy in The Research Institute at Nationwide Children’s, the programs include interviews with authors of recent scientific publications discussing how their work improves understanding of inherited neuromuscular diseases and what their findings might mean for treatment.
The initial programs include:
- Jerry Mendell, MD, director of the Center for Gene Therapy in The Research Institute at Nationwide Children’s Hospital and professor of Pediatrics and Pathology at The Ohio State University College of Medicine, discusses gene transfer of follistatin and the implications for muscle disease.
- Brian Kaspar, PhD, an investigator in the Center for Gene Therapy in The Research Institute at Nationwide Children’s and assistant professor of Pediatrics at Ohio State, explains exciting new results in animal models of gene transfer in spinal muscular atrophy.
- Kate Bushby, MBChB, MD, professor of Neuromuscular Genetics at Newcastle University (UK), discusses the recent publication of care standards in Duchenne muscular dystrophy (DMD). These standards address the diagnosis and management of DMD, including pharmacologic, orthopedic and psychological management. Dr. Bushby is also the coordinator of TREAT-NMD (Translational Research in Europe – Assessment and Treatment of Neuromuscular Diseases), a European network focusing on bringing advances in neuromuscular disease treatment rapidly and effectively to patients.
“There is a lot of exciting work going on in the field of neuromuscular disease, and for patients and their families, it may be hard to get access to information about new results,” Dr. Flanigan said. “Our goal in offering this monthly podcast is to provide a way for people affected by the muscular dystrophies and related disorders to hear directly from top researchers about their latest results. It’s my job to converse in understandable terms with these researchers about what is useful or exciting in their work.”
Patients and their families are eager to find reliable information, especially about what new therapies are entering trials. These monthly podcasts will provide reliable information directly from leading scientists and physicians in the field to empower patients to take the information they learn into their own clinics to discuss with their doctors. The podcasts also serve to provide reliable information to primary care physicians who often have the most contact with patients who have neuromuscular disorders.
“Through these podcasts, I think we can reinforce the hope shared by all families, and let them know that many pathways that may lead to meaningful treatments are being explored,” Dr. Flanigan added.
Dr. Flanigan’s primary research interest is in the genetic and molecular characterization of inherited neuromuscular diseases and the development of therapies directed toward these diseases.
Neuromuscular Disorders at Nationwide Children’s Hospital is comprised of a multidisciplinary staff that diagnoses and treats disorders affecting peripheral nerves and muscles in children. Scientists in the Center for Gene Therapy in The Research Institute at Nationwide Children’s investigate and employ the use of gene and cell-based therapeutics for prevention and treatment of neuromuscular and neurodegenerative diseases, lysosomal storage disorders, ischemia and re-perfusion injury, neonatal hypertension, cancer and infectious diseases.