Top sponsors: Children's Hospital Medical Center, Cincinnati (5) · Assistance Publique - Hôpitaux de Paris (4) · JCR Pharmaceuticals Co., Ltd. (4) · Denali Therapeutics Inc. (3) · Hospices Civils de Lyon (3)

Narrower conditions

Active trials

• Rett Syndrome 30
• Fragile X Syndrome 25
• Mucopolysaccharidosis II 22
• Adrenoleukodystrophy 19
• Creatine deficiency, X-linked 5
• Pyruvate Dehydrogenase Complex Deficiency Disease 5
• Allan-Herndon-Dudley syndrome 4
• Glycogen Storage Disease Type IIb 4
• Menkes Kinky Hair Syndrome 3

• All Trials
• Recent Changes
• New entrants

Data updated daily from ClinicalTrials.gov.