Effects of early application of PEG-rhGH on neurodevelopment and cognitive function in children with Combined methylmalonic acidemia and homocystinuria Effects of early application of PEG-rhGH on neurodevelopment and cognitive function in children with ...
Effects of early application of PEG-rhGH on neurodevelopment and cognitive function in children with Combined methylmalonic acidemia and homocystinuria Effects of early application of PEG-rhGH on neurodevelopment and cognitive function in children with ...
A Multiple Ascending Dose Study of ACN00177 (Pegtarviliase) in Subjects With CBS Deficiency
A Phase 1/2 Multiple Ascending-Dose Study in Subjects With Homocystinuria Due to Cystathionine ß-Synthase (CBS) Deficiency to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics of ACN00177 A Phase 1/2 Multiple Ascending-Dose Study in Subjects With Homocystinuria Due to Cystathionine ß-Syn ...
Homocystinuria Due to Cystathionine Beta-Synthase Deficiency
Pegtibatinase as an Enzyme Therapy for Patients With Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (COMPOSE) Pegtibatinase as an Enzyme Therapy for Patients With Homocystinuria Caused by Cystathionine Beta-Syn ...
A Double Blind, Randomized, Placebo-controlled, Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Effects on Clinical Outcomes of Pegtibatinase (TVT-058), Administered Subcutaneously in Patients With Cystathionine Beta-Synthase Deficient Homocystinuria (COMPOSE) A Double Blind, Randomized, Placebo-controlled, Phase 1/2 Study to Assess the Safety, Tolerability, ...