Neuromuscular Program

Early treatment is crucial for better outcomes in progressive neuromuscular disorders. With the establishment of Arizona's newborn screening for Spinal Muscular Atrophy (SMA), this initiative has enabled Phoenix Children’s to provide same-day genetic notification and next-day evaluation and protocols to ensure early intervention.

Innovative Treatments for Pediatric Neuromuscular Disorders

The treatment of pediatric neuromuscular disorders involves a variety of advanced and innovative approaches. Many of these treatments are being used with patients and studied in clinical trials at Phoenix Children’s. This helps ensure that our patients have access to the most advanced and effective therapies available.

Our strategies include:

We use innovative genetic therapies to help treat neuromuscular disorders, including methods such as gene replacement, exon skipping, stop codon read-through and enhancement of protein production.

We utilize treatments aimed at preventing muscle fibrosis and inflammation, as well as enhancing muscle growth and strength. These interventions help to improve overall muscle function and prevent further degeneration.

ERT is a type of treatment used for certain pediatric neuromuscular diseases where enzyme deficiency is a key factor in the disease process. This therapy aims to replace the deficient enzyme and restore its function within cells where it is needed.

Using treatments that help improve the function of the heart and the lungs helps us address the many different impacts neuromuscular disorders can have on the body.

Altering immunity to prevent the progression of these disorders is another vital approach. By working with the immune system, we aim to reduce disease progression and improve long-term outcomes.

Our Nationally Recognized Program

Our Neuromuscular Program allows us to provide timely, compassionate and comprehensive care for complex multisystem disorders, bringing together neuromuscular experts in neurology, cardiology, pulmonology, genetics, social services, nutrition, physical therapy, clinical research and clinical care coordination, all in a single visit. Our program is nationally recognized and supported by the following organizations:

  • Cure SMA Regional Treatment Site: Cure SMA has developed a national network of treatment centers to establish standards of care, a registry to track outcomes, and has developed programs to prepare institutions for clinical SMA research. Because of our treatment efforts, Phoenix Children’s has been designated a CURE SMA REGIONAL SITE.
  • PPMD Certified Duchenne Center: Parent Project Muscular Dystrophy awarded their renowned Certified Duchenne Care Center (CDCC) Program pediatric certification to Phoenix Children's. This addition to the CDCC Program was the first certification in the state of Arizona.
  • Muscular Dystrophy Association: As a certified Muscular Dystrophy Association Care Center, Phoenix Children’s is dedicated to expert clinical care and medical research for children with neuromuscular diseases. Our robust multidisciplinary team meets the unique needs of each child while providing the latest standards of care. We are proud of this partnership with the Muscular Dystrophy Association which allows us to support our neuromuscular community.