Jim Willson, a patient with spinal muscular atrophy (SMA) who is currently in a clinical trial for risdiplam (Evrysdi), gives advice to physicians and patients thinking about being part of a clinical trial.
SMA is a genetic disease that affects motor neurons in the spinal cord and control of muscle movement. It is caused by a mutation in the survival motor neuron gene 1 (SMN1) which affects nerve cells and leads to loss of muscle function.
Mr. Willson advises physicians to do their own research so they are aware of the options available to their patients with rare diseases. He advises other patients to be their own ally by researching available options and making an informed decision based on what their physicians tell them.
To learn more about this and other rare neuromuscular disorders, visit checkrare.com/diseases/musculoskeletal-diseases/