The Dystonia Coalition Natural History and Biospecimen Repository for Isolated Dystonias

A Phase 1 Study To Evaluate Escalating Doses Of A Vaccine-Based Immunotherapy Regimen For Prostate Cancer (PrCa VBIR)

Mapping the Phenotype in Adults With Phelan-McDermid Syndrome

ABI-009, an mTOR Inhibitor, for Patients With Severe Pulmonary Arterial Hypertension

The Safety and Efficacy of a Tumor Necrosis Factor Receptor Fusion Protein on Uveitis Associated With Juvenile Rheumatoid Arthritis

Dabrafenib and Trametinib in Treating Patients With Erdheim Chester Disease and BRAF Mutation

Safety and Durability of Sirolimus for Treatment of LAM

Safety Study of Unlicensed, Investigational Cord Blood Units Manufactured by the NCBP for Unrelated Transplantation

Androgen Reduction in Congenital Adrenal Hyperplasia, Phase 1

Blepharospasm Tools

Study of the Efficacy and Safety of Lonafarnib / Ritonavir With and Without Pegylated Interferon -Alfa-2a

Study of Gene Therapy Using a Lentiviral Vector to Treat X-linked Chronic Granulomatous Disease

Evaluation of the Safety and Efficacy of Ang-(1-7) to Enhance Cognitive Function in Participants Undergoing CABG

A Study of AK002 in Patients With Eosinophilic Gastritis and/or Eosinophilic Gastroenteritis

Androgen Reduction in Congenital Adrenal Hyperplasia