In addition to developing novel therapies, Dr. Zaidman practices a whole-patient approach to caring for children with neuromuscular disease and their families. He and his team treat the disease while also addressing its physical, emotional, and social consequences.
Our patients have access to leading global experts in pediatric neuromuscular diseases. As early adopters of new therapies, we work with other specialists in our combined clinic to ensure complete care for our patients and their families.
Washington University in Saint Louis is a major site in neuromuscular disease research. We participated in pivotal clinical trials and expanded access programs for numerous therapeutics that are now clinically approved products, including Zolgensma, Spinraza, Risdiplam, Exondys 51, Viltolarsen, Amondys 45, and Lumizyme, among others. As a result, our patients benefit from the knowledge and experience gained in caring for subjects in these trials. Our accomplished high-cost drug team has successfully been among the first in the country to procure newly approved advanced therapeutics for our patients.
The treatment landscape for neuromuscular diseases is changing rapidly, and we are quick to include new advanced therapeutics in our practice. Some examples include:
- Spinal muscular atrophy therapeutics - Zolgensma, Spinraza, Risdiplam
- Duchenne muscular dystrophy therapeutics - Emflaza, Exondys 51, Vyondys 53, Viltolarsen, Amondys 45
- Pompe disease therapeutics - Lumizyme, Nexviazyme
