Clinical Trials

A Randomized Study of BPN14770 in Male Adolescents (Aged 12 to < 18 Years) With Fragile X Syndrome

Amgen Osteogenesis Imperfecta Study

Assessment of Potential for Chronic Liver Injury in Participants Treated With Epidiolex (Cannabidiol) Oral Solution

The Balance Study

Cardiopulmonary Outcomes in Osteogenesis Imperfecta (BBD7708)

Characterization of the Natural History of Leukoencephalopathy with Brainstem and Spinal Cord Involvement and Lactate Elevation

Does Your Child Have Epilepsy?

Explore our Fragile X Syndrome Clinical Research Trial

Forward-March Fragile X Study

Guanfacine for Hyperactivity in Children with Down Syndrome (HYP01; HYPEbeGONE_DS)

LIFE-DSR

A Long-term Observational Study Evaluating Exon-Skipping Therapies in Patients with Duchenne Muscular Dystrophy (EVOLVE)

Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD)

Multimodal brain imaging of the neural effects of methylphenidate in patients with ADHD