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CBS Deficiency
Clinical trial pipeline · Data from ClinicalTrials.gov
See which CBS Deficiency trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
See which CBS Deficiency 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 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…
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…
Homocystinuria caused by Cystathionine Beta-Synthase (CBS) Deficiency is a rare autosomal-recessive metabolic condition characterized by an excess of homocystei…