In 2010, the Center for Gene Therapy at Nationwide Children’s Hospital 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. Now, these podcasts will be available for users on iTunes and at 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.
New programs available for download on iTunes include:
- Dr. Arthur Burghes, professor of Molecular and Cellular Biochemistry, Molecular Genetics and Neurology, The Ohio State University, discusses antisense oligomer treatment in an SMA mouse model
- Dr. Steve Wilton, professor, Australian Neuromuscular Research Institute, The University of Western Australia, discusses antisense-induced exon skipping.
- Dr. Denis Guttridge, associate professor, Molecular Virology, Immunology and Medical Genetics, The Ohio State University, discusses NF-kB therapy for Duchenne Muscular Dystrophy.
- Dr. Jill Rafael-Fortney, Department of Molecular and Cellular Biochemistry, The Ohio State University, discusses the effects of Lisinopril and Spironolactone on DMD mice.
- Dr. Valérie Allamand, the Institute of Myology, Paris, France, discusses the function of selenoprotein N in muscle and its link to neuromuscular disorders.
- Dr. Paul Martin, principal investigator, Center for Gene Therapy, The Research Institute at Nationwide Children’s Hospital, discusses how the CMAH gene deletion in the mdx mouse model of Duchenne Muscular Dystrophy helps better mimic the more severe aspects of DMD.
- Dr. Carsten Bonnemann, investigator, Neurogenetics Branch, and Chief of Neuromuscular and Neurogenetic Disorders of Childhood Section, National Institutes of Neurological Disorders and Stroke, National Institutes of Health, discusses collagen VI myopathies and a novel cause of Ullrich Congenital Muscular Dystrophy.
- Dr. Scott Harper, principal investigator, Center for Gene Therapy, The Research Institute at Nationwide Children’s Hospital, discusses DUX4 as a potential candidate gene for Facioscapulohumeral Muscular Dystrophy.
- Dr. Jerry Mendell, director, Center for Gene Therapy, The Research Institute at Nationwide Children’s Hospital, discusses dystrophin immunity in Duchenne Muscular Dystrophy.
- Dr. Craig McDonald, chair and professor of Physical Medicine and Rehabilitation and Professor of Pediatrics at UC Davis, discusses the six-minute walk test as an outcome measure in DMD.
- Dr. Annemieke Aartsma-Rus, DMD Genetic Therapy Group, Dept. of Human Genetics, Leiden University Medical Center, The Netherlands, discusses progress in aatisense Oligonucleotide therapies.
- Dr. Federica Montanaro, principal investigator, Center for Gene Therapy, The Research Institute at Nationwide Children’s Hospital, discusses cardiomyopathy in Becker Muscular Dystrophy.
Podcasts from previous months have also been uploaded to iTunes and are available for download.
“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,” said Dr. Flanigan, also a professor of Pediatrics and Neurology at The Ohio State University College of Medicine. “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. With these podcasts available on iTunes, patients and their families have access to this information at their fingertips. These monthly podcasts 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 – particularly muscular dystrophies – and in 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.