Records containing: Lysosomal Acid Lipase Deficiency

This is the wording recorded or indexed by the source. An agency index term is not necessarily an authorised indication. Co-listed drug names are not linked to this term as a treatment relationship.

All FDA submission records EMA medicine records ClinicalTrials.gov record updates ClinicalTrials.gov records with results posted

CLINICAL_TRIALS study record update · 2026-04-14
A Multicenter Real-world Observational Study of the Prevalence, Diagnostic Pathways, and Clinical Characteristics of Lysosomal Acid Lipase Deficiency in Pediatric and Adolescent Risk Groups in the Russian Federation (HELIOS)
CLINICAL_TRIALS study record update · 2026-03-06
An Observational Disease and Clinical Outcomes Registry of Patients With Lysosomal Acid Lipase (LAL) Deficiency
CLINICAL_TRIALS study record update · 2025-09-12
ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
CLINICAL_TRIALS study record update · 2025-08-12
Universal Genomic Newborn Screening in the Wallonia-Brussels Federation: Baby Detect
CLINICAL_TRIALS study record update · 2025-04-04
Early Check: A Collaborative Innovation to Facilitate Pre-Symptomatic Clinical Trials in Newborns
Organize your saved records without ads with Plus.
CLINICAL_TRIALS study record update · 2022-07-29
Prevalence and Mutation Rate of Lipa Gene in LIPIGEN Subjects With Clinical Diagnosis of FH
CLINICAL_TRIALS study record update · 2020-03-09
Metabolic Effects of Very Low Carbohydrate Ketogenic Diet in Subjects With Severe Obesity
CLINICAL_TRIALS study record update · 2019-11-18
A Phase 2, Open Label, Multicenter Study to Evaluate the Safety, Tolerability, Efficacy, and Pharmacokinetics of Sebelipase Alfa in Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency
CLINICAL_TRIALS study record update · 2019-01-15
A Multi-Center, Open-Label Study of Sebelipase Alfa in Patients With Lysosomal Acid Lipase Deficiency
CLINICAL_TRIALS study record update · 2018-12-11
An Open-Label Multicenter Study to Evaluate the Safety, Tolerability and Pharmacokinetics of SBC-102 in Adult Participants With Liver Dysfunction Due to Lysosomal Acid Lipase Deficiency
CLINICAL_TRIALS study record update · 2017-06-16
SCREENING FOR LYSOSOMAL ACID LIPASE DEFICIENCY AS THE UNDERLYING SOURCE OF HEPATIC INJURY IN PEDIATRIC PATIENTS WITH EVIDENCE OF ABNORMAL CLINICAL OR BIOCHEMICAL TESTS (DETECT)
CLINICAL_TRIALS study record update · 2016-07-20
An Observational Study of the Clinical Characteristics and Disease Progression of Patients With Lysosomal Acid Lipase Deficiency/Cholesteryl Ester Storage Disease Phenotype
CLINICAL_TRIALS study record update · 2016-06-27
A Retrospective Natural History Study of Patients With Lysosomal Acid Lipase Deficiency/Wolman Phenotype
CLINICAL_TRIALS study record update · 2016-06-08
AN EXPANDED ACCESS PROTOCOL FOR SEBELIPASE ALFA FOR PATIENTS WITH LYSOSOMAL ACID LIPASE DEFICIENCY
CLINICAL_TRIALS study record update · 2016-05-24
A Study to Identify the Frequency of Lysosomal Acid Lipase Deficiency in At-Risk Patient Populations
CLINICAL_TRIALS study record update · 2016-05-09
An Open Label Multicenter Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 in Adult Subjects With Liver Dysfunction Due to Lysosomal Acid Lipase Deficiency Who Previously Received Treatment in Study LAL-CL01
CLINICAL_TRIALS study record update · 2016-04-18
An Open Label, Multicenter, Dose Escalation Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of SBC-102 (Sebelipase Alfa) in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency
CLINICAL_TRIALS study record update · 2016-04-18
A Multicenter, Randomized, Placebo-controlled Study of SBC-102 in Patients With Lysosomal Acid Lipase Deficiency
CLINICAL_TRIALS study record update · 2013-08-13
Identification of Undiagnosed Lysosomal Acid Lipase Deficiency