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Hereditary Diseases
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Hereditary Diseases trials you may qualify forClinical trial pipeline · Data from ClinicalTrials.gov
See which Hereditary Diseases trials you may qualify forThe Duchenne Registry is an online, patient-report registry for individuals with Duchenne and Becker muscular dystrophy and carrier females. The purpose of the…
The goal of the study is to determine effects of sensorimotor technique (SMT) program with short foot exercises on navicular drop and foot posture in school goi…
ARCT-032-02 is a Phase 2, open-label, multicenter, multiple-ascending dose study of ARCT-032 in adults with CF who are not eligible for CFTR modulator therapy o…
The purpose of this study is to measure efficacy and safety of pegtibatinase treatment compared with placebo in participants with classical HCU receiving standa…
In this study, ANG003, a pancreatic enzyme replacement therapy (PERT; commonly called "enzymes"), is being investigated as a potential treatment for exocrine pa…
This is a multicenter, open-label, non-randomized study to investigate the safety, tolerability, and efficacy of a single intravenous (IV) infusion of SGT-003 i…
The purpose of the study is to assess the safety and tolerability of bilateral subretinal delivery of adeno-associated virus vector with a serotype 5 capsid hum…
Individuals with Down syndrome (DS) live with visual deficits due, in part, to elevated levels of higher-order optical aberrations (HOA). HOAs are distortions/a…