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Classical Homocystinuria
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Classical Homocystinuria trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
See which Classical Homocystinuria trials may be worth asking aboutNorth America
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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 goal of this long-term extension (LTE) study is to evaluate the safety and efficacy of pegtibatinase in patients with classical homocystinuria (HCU). Patien…
Classical homocystinuria (HCU) is an autosomal recessive disorder caused by the deficiency of an enzyme cystathionine β-synthase (CβS) that affects the cataboli…