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A Phase II, Open Study to Assess Efficacy and Safety of Rigosertib in Patients With Recessive Dystrophic Epidermolysis Bullosa Associated Locally Advanced/Metastatic Squamous Cell Carcinoma

A Non-Interventional Pilot Study to Explore the Role of Gut Flora in Epidermolysis Bullosa

Evaluation of the Efficacy of Injections of Botulinic Toxin in Plantar Lesions of Patients Suffering From Localized Epidermolysis Bullosa Simplex : Double Blind Randomized Controlled Study.

Metabolic Study of Cockayne Syndrome

The Myelin Disorders Biorepository Project and Global Leukodystrophy Initiative Clinical Trials Network

Augmentation of Umbilical Cord Blood Transplantation for Inherited Metabolic Diseases With Intrathecal Administration of Human Umbilical Cord Blood-Derived Oligodendrocyte-Like Cells

Longitudinal Study of Neurodegenerative Disorders

Online Study of People Who Have Genetic Changes and Features of Autism: Simons Searchlight

Coordination of Rare Diseases at Sanford

Examination of Clinical and Laboratory Abnormalities in Patients With Defective DNA Repair: Xeroderma Pigmentosum, Cockayne Syndrome, or Trichothiodystrophy

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