Records containing: Pompe Disease (Late-onset)
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.
Follow recorded terms in Drug Alerts for Android. Free includes unlimited local condition follows and an on-open recent-record watchlist. Plus adds no ads and personal organization tools.
All
FDA submission records
EMA medicine records
ClinicalTrials.gov record updates
ClinicalTrials.gov records with results posted
-
CLINICAL_TRIALS study record update · 2026-04-16A Sub-registry to Observe the Effect of Alglucosidase Alfa or Avalglucosidase Alfa Treatment on Pregnancy and Infant Growth in Women With Pompe Disease
-
CLINICAL_TRIALS study record update · 2026-04-09A Single-Arm, Open-Label, Dose-Escalation Study to Evaluate the Safety, Tolerability and Efficacy of a Single Intravenous Infusion of AB-1009 in Adult Participants With Late Onset Pompe Disease (LOPD)
-
CLINICAL_TRIALS study record update · 2026-04-08A Study to Evaluate Seroprevalence of Antibodies to AAV8 and Assessment of Biomarkers in Patients With Late-Onset Pompe Disease
-
CLINICAL_TRIALS study record update · 2026-03-17Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults withNeuromuscular Disease
-
CLINICAL_TRIALS study record update · 2026-02-02A Phase 1/2, Open-Label, Ascending-Dose Clinical Study to Evaluate the Safety and Preliminary Efficacy of AT845, an AAV8-Delivered Gene Transfer Therapy in Patients With Late Onset Pompe Disease
- Organize your saved records without ads with Plus.
-
CLINICAL_TRIALS study record update · 2026-01-12A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease (LOPD)
-
CLINICAL_TRIALS study record update · 2025-10-27An Open-label Study of the Safety, Pharmacokinetics, Efficacy, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Pediatric Subjects Aged 0 to < 18 Years With Late-onset Pompe Disease
-
CLINICAL_TRIALS study record update · 2025-09-22Developing a Management Approach for Patients With "Late-Onset" Pompe Disease GAA Variant Identified by Newborn Screening
-
CLINICAL_TRIALS study record update · 2025-08-11Using Carbon-13 Magnetic Resonance Spectroscopy to Assess Whether High Glycogen Levels in Skeletal Muscle of Patients With Glycogen Storage Diseases is a Prelude for Muscle Damage.
-
CLINICAL_TRIALS study record update · 2025-07-03A Multi-centered, Single Arm, Open Labeled, Study to Evaluate the Safety, Tolerability, and Efficacy of an Adeno-associated Virus Vector Expressing the Human Acid Alpha-glucosidase (GAA) Transgene Intravenous Injection in Patients With Late-onset Pompe Disease
-
CLINICAL_TRIALS study record update · 2025-03-12Multispectral Optoacoustic Tomography for Translational Molecular Imaging in Pompe Disease
-
CLINICAL_TRIALS study record update · 2024-11-27Phase 1/2, Dose-escalation Study to Evaluate the Safety, Tolerability and Efficacy of a Single Intravenous Infusion of SPK-3006 in Adults With Late-onset Pompe Disease
-
CLINICAL_TRIALS study record update · 2024-08-28Open Label, Single-center Pilot Study to Investigate Alglucosidase Alfa (20 mg/kg) Frequency Reduction From 2 to 4 Weeks in a Subgroup of Elderly Patients With Late-onset Pompe Disease (TRIPOD-Study)
-
CLINICAL_TRIALS study record update · 2023-12-21A Single-arm, Open-label, Single-dose Study to Evaluate the Safety, Tolerability, and Efficacy of CRG003 Injection in the Treatment of Late Onset Pompe Disease
-
CLINICAL_TRIALS study record update · 2023-10-10Pompe & Pain - Observational Study to Assess Musculoskeletal Pain in Late-onset Pompe Disease (LOPD)
-
CLINICAL_TRIALS study record update · 2023-09-11A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease Compared With Alglucosidase Alfa/Placebo
-
CLINICAL_TRIALS study record update · 2023-07-19Efficacy and Feasibility of Inspiratory Muscle Training (IMT) in Adult People With Pompe Disease: a Multicentre, Cross-over Randomized Control Trial (RCT)
-
CLINICAL_TRIALS study record update · 2023-03-24A Multi-Center, Low-Interventional Study With a Retrospective Component in Participants With Late-Onset Pompe Disease
-
CLINICAL_TRIALS study record update · 2022-10-24Multicenter Non-Drug Screening Study to Determine the Frequency of Duchenne Muscular Dystrophy and Late-onset Pompe Disease in Children With Unexplained Transaminase Elevation
-
CLINICAL_TRIALS study record update · 2022-08-24Nociceptive Pain in Neuromuscular Disorders - Low Interventional Pilot Study to Assess Musculoskeletal Pain in Neuromuscular Disorders (NMD)
-
CLINICAL_TRIALS study record update · 2022-08-17Phase II Clinical Trial of Clenbuterol in Adult Patients With Pompe Disease Stably Treated With Enzyme Replacement Therapy
-
CLINICAL_TRIALS study record update · 2019-07-02Screening for Eligibility to Participate in a Clinical Trial of Gene Therapy for Late-onset Pompe Disease
-
CLINICAL_TRIALS study record update · 2014-02-06Single-center, Open-label Study of Safety, Pharmacokinetics and Efficacy of rhGAA in Patients With Late-Onset Pompe Disease
-
CLINICAL_TRIALS study record update · 2014-02-05Prospective, Open-label, Single-arm, Exploratory Study of the Effect and Safety of rhGAA in Patients With Advanced Late-onset Pompe Disease Who Are Receiving Respiratory Support
-
CLINICAL_TRIALS study record update · 2010-07-23Randomized, Double-Blind, Placebo-Controlled Study of the Safety, Efficacy and Pharmacokinetics of Myozyme in Patients With Late-Onset Pompe Disease.