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A Phase I, Randomized, Double-Blind, Placebo-Controlled, Single Ascending Dose (SAD) Study Including a Food Interaction Study, Followed by a Multiple Ascending Dose (MAD) Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamic Profile of Progerinin in Healthy Volunteers

Metabolic Study of Cockayne Syndrome

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

Phase I/II Trial of Everolimus in Combination With Lonafarnib in Progeria

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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