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Homocystinuria
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Homocystinuria trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
See which Homocystinuria trials may be worth asking aboutNorth America
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Trials may have sites in multiple countries. Filtering shows any trial with at least one site in the selected country.
The purpose of this study is to measure efficacy and safety of pegtibatinase treatment compared with placebo in participants with classical HCU receiving standa…
Patients, parents of young / handicapped patients, and experts will be interviewed to collect contents relevant for HrQoL in CBS deficiency. Based on these data…
The purpose of the study is to characterize the clinical course of homocystinuria in pediatric and adult patients aged 1 to 65 years under current clinical mana…
General aim of the study is the improvement of the clinical knowledge of ultra-rare inherited metabolic and degenerative neurological diseases (prevalence less…
The goal of this long-term extension (LTE) study is to evaluate the safety and efficacy of pegtibatinase in patients with classical homocystinuria (HCU). Patien…
A prospective, open label, acceptability study to evaluate PKU, MSUD, HCU, TYR and GA express plus in the dietary management of 40 patients with IEM. The follow…