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Gene-based therapies

All patients seen in the neuromuscular clinic receive multidisciplinary supportive care, which includes assessments of physical and occupational therapy needs, braces, equipment, and nutrition counseling. For some conditions, patients may be followed by lung or heart specialists and receive respiratory therapies or medications for heart function. Orthopedics is also available to address scoliosis and other musculoskeletal deformities. Our social worker and nurse program coordinator work to coordinate between specialists and connect families to financial, educational, and community resources. Our palliative care team is available as well to provide additional support as needed.

An expanding number of neuromuscular conditions may be treated with targeted, genetic therapies. We provide the following treatments for appropriate patients.

Duchenne Muscular Dystrophy

  • Eteplirsen (Exondys51)
  • Golodirsen (Viondys53)
  • Viltolarsen (Viltepso)

Spinal Muscular Atrophy

  • Gene replacement therapy: Onasemnogene abeparvovec-xioi (Zolgensma)
  • Nusinersen (Spinraza)
  • Risdiplam (Evrysdi)

Pompe Disease

  • Alglucosidase alfa (Lumizyme)

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