Pompe Disease
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS trial_result · 2021-06-11Clinical Trial Update: Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease [Phase 4]
Phase 4 pharmacokinetic study of alglucosidase alfa in Pompe disease patients has completed and results were posted in 2021.
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CLINICAL_TRIALS trial_result · 2021-04-08Clinical Trial Update: Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease [Phase 3]
Phase 3 trial comparing avalglucosidase alfa to alglucosidase alfa in late-onset Pompe disease patients who have not been treated before, wi…
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CLINICAL_TRIALS trial_result · 2020-10-12Clinical Trial Update: Immune Tolerance Induction Study [Phase 4]
Phase 4 trial update for Immune Tolerance Induction in Pompe disease was terminated with results posted in 2020.
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FDA approval · 2020-02-18FDA Approved: LUMIZYME — Supplemental Application (Genzyme Corporation)
The FDA approved a supplemental application for Lumizyme (alglucosidase alfa) by Genzyme Corporation.
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CLINICAL_TRIALS trial_result · 2018-08-17Clinical Trial Update: Drug-drug Interaction Study [Phase 2]
Amicus Therapeutics completed a Phase 2 drug–drug interaction study in Pompe disease, and the results have been posted.
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CLINICAL_TRIALS trial_result · 2018-08-17Clinical Trial Update: Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe Disease [Phase 2]
A Phase 2 safety study of AT2220 (duvoglustat) for Pompe disease was terminated, with results posted in 2018.
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CLINICAL_TRIALS trial_result · 2018-06-11Clinical Trial Update: Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease [Phase 1, Phase 2]
BMN701 was studied in late-onset Pompe disease in a Phase 1/2 trial focused on safety, tolerability, pharmacokinetics and pharmacodynamics. …
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CLINICAL_TRIALS trial_result · 2018-05-22Clinical Trial Update: Extension Study for Patients Who Have Participated in a BMN 701 Study [Phase 2]
Extension study of BMN 701 in Pompe disease participants from a prior BMN 701 trial (Phase 2) has been terminated; results were posted in 20…
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CLINICAL_TRIALS trial_result · 2018-05-15Clinical Trial Update: A Pilot Study of Pyridostigmine in Pompe Disease [EARLY_Phase 1]
An early-phase 1 pilot study evaluating pyridostigmine for Pompe disease has been terminated.
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CLINICAL_TRIALS trial_result · 2018-02-06Clinical Trial Update: Safety and Efficacy of Albuterol in Individuals With Late-onset Pompe Disease [Phase 1, Phase 2]
Phase 1/2 trial update evaluating safety and efficacy of albuterol in individuals with Pompe disease has completed with results posted.
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CLINICAL_TRIALS trial_result · 2017-12-08Clinical Trial Update: Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies
This completed observational study evaluated subjects with Pompe disease undergoing immune modulation therapies.
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CLINICAL_TRIALS trial_result · 2017-11-17Clinical Trial Update: Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease [Phase 1, Phase 2]
A phase 1/2 clinical trial evaluating safety of a recombinant adeno-associated virus delivering acid alpha-glucosidase for Pompe disease has…
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CLINICAL_TRIALS trial_result · 2017-10-23Clinical Trial Update: Safety and Efficacy of Clenbuterol in Individuals With Late-onset Pompe Disease and Receiving Enzyme Replacement Therapy [Phase 1, Phase 2]
Clinical trial assessing safety and efficacy of clenbuterol in individuals with late-onset Pompe disease on enzyme replacement therapy (Phas…
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CLINICAL_TRIALS research · 2017-07-18Clinical Trial Update: A Long-term Study for the Outcome of Pompe Disease
A long-term clinical study assessing outcomes in patients with Pompe disease is currently recruiting.
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CLINICAL_TRIALS trial_result · 2017-05-16Clinical Trial Update: Cardiopulmonary Exercise Test to Quantify Enzyme Replacement Response in Pediatric Pompe Disease
This completed clinical trial update investigates using cardiopulmonary exercise testing to quantify the response to enzyme replacement ther…
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CLINICAL_TRIALS research · 2017-04-04Clinical Trial Update: Investigating Lysosomal Storage Diseases in Minority Groups
A clinical trial update from O & O Alpan LLC examining lysosomal storage disorders in minority populations, including Gaucher disease, Fabry…
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CLINICAL_TRIALS research · 2017-03-20Clinical Trial Update: Search for Serum/Plasma Biomarkers in Pompe's Disease
A clinical trial is investigating serum/plasma biomarkers in Pompe disease, including late-onset Pompe disease, to advance biomarker discove…
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CLINICAL_TRIALS trial_result · 2016-09-28Clinical Trial Update: Pregnancy and Birth Outcome in Women With Pompe Disease
This document reports the completion of a clinical trial examining pregnancy and birth outcomes in women with Pompe disease.
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CLINICAL_TRIALS trial_result · 2016-09-20Clinical Trial Update: Evaluation of Salbutamol as an Adjuvant Therapy for Pompe Disease [Phase 4]
Phase 4 clinical trial evaluating salbutamol as adjuvant therapy for Pompe disease has completed.
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CLINICAL_TRIALS trial_result · 2016-09-19Clinical Trial Update: Feasability and Interest of Screening for Infantile Pompe's Diseases at Birth
Completed study evaluating the feasibility and interest of newborn screening for infantile Pompe disease at birth.
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CLINICAL_TRIALS trial_result · 2016-09-16Clinical Trial Update: Prevalence of Heterozygote Mothers for Pompe's Disease Among Mothers Having Delivered in French Guiana
A completed clinical trial assessing the prevalence of heterozygote mothers for Pompe disease among women who delivered in French Guiana.
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CLINICAL_TRIALS trial_result · 2016-05-04Clinical Trial Update: Effect of Motor Development, Motor Function and Electrophysiologic Findings of IOPD Under ERT
Update on a clinical trial evaluating motor development, motor function, and electrophysiologic findings in infants with Pompe disease treat…
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CLINICAL_TRIALS trial_result · 2016-04-15Clinical Trial Update: Immune Modulation Therapy for Pompe Disease [Phase 4]
Phase 4 clinical trial update on an immune modulation therapy for Pompe disease; current status is unknown.
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CLINICAL_TRIALS trial_result · 2016-01-18Clinical Trial Update: A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease [Phase 4]
Phase 4 noninferiority trial evaluating alglucosidase alfa manufactured at 160 L vs 4000 L scales in treatment-naïve infants with Pompe dise…
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CLINICAL_TRIALS trial_result · 2015-10-27Clinical Trial Update: A Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe Disease [Phase 4]
Phase 4 clinical trial evaluating alglucosidase alfa produced at the 4000 L manufacturing scale for Pompe disease. The study was terminated …