Alpha-mannosidosis
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Sign up freeTop drugs studied for this disease
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EMA approval · 2026-03-02EMA Authorised: Lamzede (velmanase alfa) — Chiesi Farmaceutici S.p.A.
The European Medicines Agency authorised Lamzede (velmanase alfa) by Chiesi Farmaceutici S.p.A. for the treatment of alpha-mannosidosis, wit…
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CLINICAL_TRIALS trial_result · 2026-01-07Clinical Trial Update: MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis [Phase 2]
Phase 2 trial update describing allogeneic hematopoietic cell transplantation for metabolic disorders and severe osteopetrosis, with the stu…
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CLINICAL_TRIALS trial_result · 2025-05-16Clinical Trial Update: Analysis of Velmanase Alfa (Lamzede®)'s Effects in the Body of Children With Alpha-Mannosidosis Under the Age 3
This clinical trial update describes a study of Velmanase alfa (Lamzede) in children under 3 with Alpha-mannosidosis that is currently recru…
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CLINICAL_TRIALS trial_result · 2023-07-28Clinical Trial Update: Lamazym Aftercare Study [Phase 3]
Phase 3 clinical trial update for Lamazym Aftercare in Alpha-Mannosidosis has completed.
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CLINICAL_TRIALS trial_result · 2023-07-28Clinical Trial Update: Lamazym Aftercare Study FR Designed to Provide Treatment for French Patients [Phase 3]
Phase 3 clinical trial update for Lamazym Aftercare in French patients with Alpha-Mannosidosis has completed.
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FDA approval · 2023-02-16FDA Approved: LAMZEDE — Original Application (Chiesi USA, Inc.)
FDA approved Lamzede (velmanase alfa-tycv) from Chiesi USA as an original Biologics License Application for the treatment of alpha-mannosido…
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CLINICAL_TRIALS trial_result · 2021-10-26Clinical Trial Update: Trial on Safety and Efficacy of Velmanase Alfa Treatment in Pediatric Patients With Alpha-Mannosidosis [Phase 2]
Phase 2 trial update on velmanase alfa for pediatric alpha-mannosidosis completed, reporting safety and efficacy findings.
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CLINICAL_TRIALS withdrawal · 2020-12-08Clinical Trial Update: Interventional Study to Assess Efficacy and Safety of Velmanase Alfa in Patients With Alpha Mannosidosis [Phase 3]
Phase 3 interventional trial evaluating velmanase alfa in Alpha Mannosidosis was withdrawn.
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CLINICAL_TRIALS trial_result · 2020-11-20Clinical Trial Update: Evaluation of Long-term Efficacy of Treatment With Lamazym [Phase 3]
Phase 3 trial evaluating long-term efficacy of Lamazym in Alpha-Mannosidosis has completed.
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CLINICAL_TRIALS trial_result · 2020-08-03Clinical Trial Update: A Placebo-Controlled Phase 3 Trial of Repeated Lamazym Treatment of Subjects With Alpha-Mannosidosis [Phase 3]
A randomized, placebo-controlled Phase 3 trial evaluating repeated Lamazym treatment in subjects with alpha-mannosidosis has been completed.
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EMA approval · 2018-01-26New enzyme replacement therapy to treat rare genetic disorder alpha-mannosidosis in children and adults
EMA announces approval of a new enzyme replacement therapy for alpha-mannosidosis, a rare genetic disorder, for use in children and adults.
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CLINICAL_TRIALS trial_result · 2017-03-29Clinical Trial Update: Long-term Efficacy and Safety of Lamazym for the Treatment of Patients With Alpha-Mannosidosis [Phase 2]
This update summarizes the long-term efficacy and safety results from a Phase 2 study of Lamazym in patients with Alpha-Mannosidosis.
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CLINICAL_TRIALS trial_result · 2012-09-26Clinical Trial Update: Dose Finding Study of Recombinant Human Alpha-mannosidase for the Treatment of Patients With Alpha-mannosidosis [Phase 2]
Phase 2 dose-finding trial of recombinant human alpha-mannosidase for Alpha-mannosidosis; the status of the study is unknown.