Clinical Trial

The evolving landscape of potential new therapies and associated clinical trials offers great hope for children affected by rare diseases and their families. With these advancements, families must learn new terms, make a different set of complex decisions and potentially face an unanticipated grief of a new kind.  EVALUATING THE CLINICAL TRIAL OPTION presents information and a clear-eyed picture of the process through the lens of families, researchers and professionals. The more parents understand, the better equipped and empowered they will be to consider participation.