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About Clinical Trials

Clinical trials (also called studies) are used to learn about the safety and effectiveness of potential new treatments, medical devices and medical procedures, and play a vital role in the development of future medical treatments. 

Although there are many types of clinical trials, all United States-based clinical trials must conform to strict rules set by the U.S. Food and Drug Administration (FDA). These rules help protect the rights and safety of those who volunteer to take part in clinical trials.

The purpose of the Resilient study is to learn how well the study drug works and how safe the study drug is, as compared to placebo, as a potential therapy to improve muscle volume and function in children and young adults with SMA. 

dog lying down and a girl sitting on bench and boy in wheelchair

Resilient is being conducted, in conjunction with Biohaven Pharmaceuticals, by multiple sites (research clinics or hospitals) across the United States and globally, with trained and qualified medical specialists. Biohaven is a biopharmaceutical company with innovative drug candidates targeting neurological diseases. 

As always in clinical studies, participation in Resilient is completely voluntary. Participants may withdraw at any time and for any reason. 

For more information on Resilient and other clinical studies, visit

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