Angelman Clinical Trials

Finding a cure for rare diseases takes time and a lot of testing. Dr. Harry Angelman published an academic research paper in 1965 describing three children he believed had the same rare disease. It took more than 20 years for researchers to discover that a problem with the 15th chromosome caused the children’s symptoms and another 12 years to isolate the Angelman Syndrome gene, UBE3A.

Identifying the AS gene in 1997 completely changed the game for the Angelman community. It gave scientists a concrete place to start their research projects and a way to see if their experiments worked. Now, more than 20 years later, the medical professionals are ready to see if their treatments really help our loved ones. How? Through clinical trials.

Participating in clinical trials is the next step toward finding a cure for AS, and we encourage you to learn more about the opportunity. We designed this site as a trusted source of information about the process and hope it answers your questions.