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X-Linked Genetic Diseases clinical trials recruiting in the U.S.
5 studies for X-Linked Genetic Diseases are recruiting participants at sites across the United States.
By state
Recently updated studies
Recruitment and Apheresis Collection of Peripheral Blood Hematopoietic Stem Cells, Mononuclear Cells and Granulocytes
RecruitingObservational studyHealthy volunteers welcome
Ages 18 to 70 · All sexes · Updated Sep 22, 2026 · NCT00001405Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)
RecruitingObservational study
7 Months – 15 Months · All sexes · Updated Aug 31, 2026 · NCT06805695Rett Syndrome Registry
RecruitingObservational study
Ages 0 to 99 · All sexes · Updated Jun 30, 2026 · NCT05432349Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)
RecruitingPhase 3
Ages 4 to 18 · Men · Updated May 27, 2026 · NCT07608432To Investigate the Efficacy of Treatment With Oral NA-921 (Bionetide) Versus Placebo in Females With Rett Syndrome
RecruitingPhase 3
Ages 5 to 20 · Women · Updated Mar 27, 2026 · NCT06840496From ClinicalTrials.gov, data retrieved Oct 2, 2026. Each study sets its own eligibility; the study team decides who can join.