Children's Hospital Colorado

Cutting-edge Therapies for Neuromuscular Disorders (S4:E33)

Transformative therapies, such as immunotherapy for leukemia and other cancers and new drug treatments for cystic fibrosis, have dramatically improved patients’ lives. Innovative new therapies are also transforming care for the treatment of children with neuromuscular diseases.

Listen to pediatric experts discuss neuromuscular disorder therapies

In this episode, Susan Apkon, MD, and Julie Parsons, MD, join us to discuss the impact that cutting-edge therapies for neuromuscular disease have had on improving the outcome and quality of life for patients.

Dr. Apkon is the Fischahs Endowed Chair of Pediatric Rehabilitation Medicine at Children’s Hospital Colorado, Chief of Pediatric Rehabilitation and Vice-Chair of Physical Medicine and Rehabilitation at the University of Colorado School of Medicine.

Dr. Parsons is the Harberfeld Family Endowed Chair in Pediatric Neuromuscular Disorders at Children’s Colorado and Professor of Clinical Pediatrics and Neurology at the University of Colorado School of Medicine.

In this episode, our experts discuss:

  • Recent advances in screening and treatment of spinal muscular atrophy (SMA
  • Treatment of SMA with Spinraza® (nusinersen)
  • Diagnosis and management of Duchenne muscular dystrophy
  • New FDA-approved drugs and clinical trials for Duchenne
  • Importance of coordinated multidisciplinary care for patients with neuromuscular diseases
  • Systemic challenge of providing access to novel, expensive treatments

Treatment of neuromuscular disorders at Children’s Colorado

The Neuromuscular Program for Muscle Disorders at Children's Colorado specializes in the evaluation, diagnosis and care of infants, children, teens and young adults with neuromuscular disorders. Our multidisciplinary neuromuscular team provides cutting-edge therapies and is involved in a variety of clinical trials to deliver the most advanced treatments to our patients.

Refer a patient to Children’s Colorado.