Challenges of developing and conducting clinical trials in rare disorders - Kempf, Lucas; Goldsmith, Jonathan C.; Temple, Robert (American journal of medical genetics. Part A, 2018)
Keywords: Animals; Biomarkers; Children; Clinical trials; Clinical Trials as Topic - legislation & jurisprudence; Clinical Trials as Topic - methods; Disease; Drug Development; Drug Evaluation, Preclinical; Food and Drug Administration (FDA); Humans; orphan drugs; Patient Selection; Patients; Product Surveillance, Postmarketing; rare disease; Rare diseases; Rare Diseases - diagnosis; Rare Diseases - therapy; rare disorders; regulation; Research Design; trial design; rare disease; Food and Drug Administration (FDA); regulation; trial design; orphan drugs; rare disorders