Lysosomal Acid Lipase Deficiency
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CLINICAL_TRIALS research · 2026-04-14Clinical Trial Update: Lysosomal Acid Lipase Deficiency in Risk Groups
AstraZeneca is recruiting participants for a clinical trial addressing lysosomal acid lipase deficiency in at-risk groups.
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EMA approval · 2026-03-24EMA Authorised: Kanuma (sebelipase alfa) — Alexion Europe SAS
The EMA authorised Kanuma (sebelipase alfa) for an inborn error of lipid metabolism. It was granted orphan medicine status with accelerated …
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CLINICAL_TRIALS research · 2026-03-06Clinical Trial Update: Lysosomal Acid Lipase (LAL) Deficiency Registry
Clinical trial registry update describing a Lysosomal Acid Lipase (LAL) deficiency registry that is currently recruiting to collect data on …
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FDA approval · 2024-07-10FDA Approved: KANUMA — Supplemental Application (Alexion Pharmaceuticals, Inc.)
FDA approved a supplemental application for KANUMA (sebelipase alfa) by Alexion Pharmaceuticals, Inc.
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FDA approval · 2021-11-19FDA Approved: KANUMA — Supplemental Application (Alexion Pharmaceuticals, Inc.)
FDA approved a supplemental application for KANUMA (sebelipase alfa) from Alexion Pharmaceuticals, Inc.
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CLINICAL_TRIALS trial_result · 2019-11-18Clinical Trial Update: Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency [Phase 2]
Phase 2 clinical trial in infants with rapidly progressive lysosomal acid lipase deficiency has been terminated; results posted in 2019.
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CLINICAL_TRIALS trial_result · 2019-01-15Clinical Trial Update: Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency [Phase 2]
Phase 2 trial assessing safety and efficacy of sebelipase alfa in lysosomal acid lipase deficiency has completed with results posted.
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CLINICAL_TRIALS trial_result · 2018-12-11Clinical Trial Update: Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Participants With Lysosomal Acid Lipase Deficiency [Phase 1, Phase 2]
Phase 1/2 trial update evaluating safety, tolerability, and pharmacokinetics of SBC-102 (Sebelipase alfa) in adults with lysosomal acid lipa…
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CLINICAL_TRIALS trial_result · 2017-06-16Clinical Trial Update: Screening for Lysosomal Acid Lipase Deficiency
The screening trial for lysosomal acid lipase deficiency (NCT02926872) sponsored by Alexion Pharmaceuticals has been terminated.
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CLINICAL_TRIALS research · 2016-08-02Clinical Trial Update: Assessment of the Prevalence of Lysosomal Acid Lipase Deficiency in Patients Waiting for a Liver Transplant.
Clinical trial update assessing how common lysosomal acid lipase deficiency is among patients awaiting liver transplantation.
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CLINICAL_TRIALS research · 2016-08-01Clinical Trial Update: Assessement of the Prevalence of Lysosomal Acid Lipase Deficiency in Liver Post-transplant Patients
This clinical trial update evaluates the prevalence of lysosomal acid lipase deficiency in patients who have undergone liver transplantation…
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CLINICAL_TRIALS research · 2016-07-20Clinical Trial Update: An Observational Study of Patients With Lysosomal Acid Lipase Deficiency/Cholesteryl Ester Storage Disease Phenotype
An observational study of patients with lysosomal acid lipase deficiency and the cholesteryl ester storage disease phenotype has completed.
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CLINICAL_TRIALS research · 2016-06-27Clinical Trial Update: A Retrospective Natural History Study of Patients With Lysosomal Acid Lipase Deficiency/Wolman Phenotype
A completed retrospective natural history study of patients with Lysosomal acid lipase deficiency and Wolman disease, documenting clinical f…
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CLINICAL_TRIALS withdrawal · 2016-06-08Clinical Trial Update: An Expanded Access Protocol for Sebelipase Alfa for Patients With Lysosomal Acid Lipase Deficiency
Expanded access protocol for sebelipase alfa to treat lysosomal acid lipase deficiency; the protocol is no longer available.
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CLINICAL_TRIALS trial_result · 2016-05-24Clinical Trial Update: A Study to Identify and Characterize LAL-D Patients in High-risk Populations
A terminated clinical trial update from Alexion investigating identification and characterization of patients with lysosomal acid lipase def…
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CLINICAL_TRIALS trial_result · 2016-05-09Clinical Trial Update: Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 (Sebelipase Alfa) in Adult Subjects With Lysosomal Acid Lipase Deficiency [Phase 2]
Phase 2 extension study evaluating long-term safety, tolerability, and efficacy of Sebelipase alfa (SBC-102) in adults with lysosomal acid l…
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CLINICAL_TRIALS trial_result · 2016-04-18Clinical Trial Update: Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency [Phase 2, Phase 3]
This clinical trial update reports Phase 2/3 results for Sebelipase alfa in children with growth failure due to lysosomal acid lipase defici…
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CLINICAL_TRIALS trial_result · 2016-04-18Clinical Trial Update: Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase Deficiency [Phase 3]
Phase 3 trial assessing safety and efficacy of acid lipase replacement in lysosomal acid lipase deficiency has completed with results posted…
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FDA approval · 2015-12-08FDA Approved: KANUMA — Original Application (Alexion Pharmaceuticals, Inc.)
The FDA approved Kanuma (sebelipase alfa) from Alexion Pharmaceuticals as the original approval for the treatment of lysosomal acid lipase d…
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EMA approval · 2015-06-26EMA fast-tracks enzyme replacement therapy for lysosomal acid lipase deficiency
EU regulators have fast-tracked an enzyme replacement therapy for lysosomal acid lipase deficiency, accelerating development and potential a…
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CLINICAL_TRIALS research · 2013-08-13Clinical Trial Update: Identification of Undiagnosed Lysosomal Acid Lipase Deficiency [NA]
The clinical trial update notes identification of undiagnosed lysosomal acid lipase deficiency and cholesterol ester storage disease within …