Clinical Trials

A Phase 4, Randomized, Double-Blind, Multicenter, Placebo-Controlled, Parallel-Group Study of the Efficacy and Safety of SPN 812 in Preschool-Age Children (4 to 5 years old) with Attention-Deficit/Hyperactivity Disorder (ADHD)

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

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

Cardiopulmonary Outcomes in Osteogenesis Imperfecta (BBD7708)

Cerebral Adrenoleukodystrophy (CALYX)

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

Genetic Causes of Intellectual Disability

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

A Phase 1-2, Double-Blind Sham-Controlled Multiple Ascending Dose Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Patients with MECP2 Duplication Syndrome (ATTUNE)

Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD)

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