Pompe Disease
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS research · 2015-08-18Clinical Trial Update: Prevalence of Pompe's Disease in Respiratory Clinics
A clinical trial update examining the prevalence of Pompe disease among patients seen in respiratory clinics.
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CLINICAL_TRIALS research · 2015-05-05Clinical Trial Update: Screening Protocol to Evaluate Acid Alpha-Glucosidase (GAA) Activity and GAA Gene Mutations in Patients With Late Onset Pompe Disease
This completed clinical trial outlines a screening protocol to measure acid alpha-glucosidase activity and GAA gene mutations in patients wi…
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CLINICAL_TRIALS withdrawal · 2015-04-13Clinical Trial Update: High Protein and Exercise Therapy Plus Nocturnal Enteral Feeding in Juvenile-onset Pompe Disease [Phase 2]
A Phase 2 clinical trial evaluating high-protein and exercise therapy with nocturnal enteral feeding for juvenile-onset Pompe disease has be…
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CLINICAL_TRIALS trial_result · 2015-04-01Clinical Trial Update: CPAP for Infantile Pompe Disease [Phase 4]
Phase 4 clinical trial update evaluating CPAP therapy for infantile Pompe disease, sponsored by National Taiwan University Hospital. The tri…
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CLINICAL_TRIALS trial_result · 2015-03-24Clinical Trial Update: Muscle Response to Enzyme Replacement Therapy in Pompe Disease
This clinical trial update reports on muscle response to enzyme replacement therapy in Pompe disease, sponsored by National Taiwan Universit…
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CLINICAL_TRIALS trial_result · 2014-12-02Clinical Trial Update: Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa [Phase 4]
Phase 4 exploratory study assessing muscle biopsy changes in patients with late-onset Pompe disease treated with alglucosidase alfa; results…
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CLINICAL_TRIALS trial_result · 2014-11-13Clinical Trial Update: Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease [Phase 2]
Phase 2 clinical trial assessing the safety and efficacy of recombinant human acid alpha-glucosidase for classical infantile Pompe disease; …
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FDA approval · 2014-08-01FDA Approved: LUMIZYME — Supplemental Application (Genzyme Corporation)
The FDA approved a supplemental application for LUMIZYME (alglucosidase alfa) submitted by Genzyme Corporation.
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FDA approval · 2014-05-22FDA Approved: LUMIZYME — Supplemental Application (Genzyme Corporation)
FDA approved a supplemental application for Lumizyme (alglucosidase alfa) from Genzyme Corporation.
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CLINICAL_TRIALS trial_result · 2014-05-13Clinical Trial Update: An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe Disease [Phase 4]
Phase 4 exploratory trial evaluating the safety and efficacy of prophylactic immunomodulatory treatment in Myozyme-naive CRIM-negative infan…
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CLINICAL_TRIALS trial_result · 2014-02-06Clinical Trial Update: Extension Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602 [Phase 2, Phase 3]
This document summarizes the completion of an extension study for infantile-onset Pompe disease patients previously enrolled in protocol AGL…
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CLINICAL_TRIALS trial_result · 2014-02-06Clinical Trial Update: A Study of rhGAA in Patients With Late-Onset Pompe Disease [Phase 2]
Phase 2 study of recombinant human acid alpha-glucosidase (rhGAA) in patients with late-onset Pompe disease has completed.
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CLINICAL_TRIALS approval · 2014-02-06Clinical Trial Update: Alglucosidase Alfa Temporary Access Program
The document notes that alglucosidase alfa has received approval for a temporary access program with marketing approval for Pompe disease an…
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CLINICAL_TRIALS trial_result · 2014-02-06Clinical Trial Update: Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Late-onset Pompe Disease [NA]
The document reports expanded access use of Myozyme (alglucosidase alfa) in patients with Pompe disease. It notes that the program status is…
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CLINICAL_TRIALS trial_result · 2014-02-06Clinical Trial Update: Study About the Evolution of Severe Late Onset Pompe Disease Patient With Pulmonary Dysfunction and Receiving Myozyme®
This clinical trial update describes the progression of a patient with severe late-onset Pompe disease and pulmonary dysfunction who is bein…
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CLINICAL_TRIALS trial_result · 2014-02-05Clinical Trial Update: A Study of the Safety and Efficacy of rhGAA in Patients With Infantile-onset Pompe Disease [Phase 2, Phase 3]
Phase 2/3 trials evaluated the safety and efficacy of recombinant human acid alpha-glucosidase (alglucosidase alfa) in infants with Pompe di…
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CLINICAL_TRIALS approval · 2014-02-05Clinical Trial Update: Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Infantile-onset Pompe Disease
This update covers expanded access use of Myozyme (alglucosidase alfa) for infantile-onset Pompe disease and notes marketing approval for th…
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CLINICAL_TRIALS trial_result · 2014-02-05Clinical Trial Update: Safety and Effectiveness Study of rhGAA in Patients With Advanced Late-Onset Pompe Disease Receiving Respiratory Support [Phase 3]
A Phase 3 trial evaluating rhGAA (alglucosidase alfa) in patients with advanced late-onset Pompe disease on respiratory support has complete…
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CLINICAL_TRIALS trial_result · 2014-02-05Clinical Trial Update: A Study of the Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II [Phase 2]
Phase 2 study evaluating the safety and pharmacokinetics of recombinant human acid alpha-glucosidase (rhGAA) in siblings with glycogen stora…
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CLINICAL_TRIALS trial_result · 2014-02-05Clinical Trial Update: Extension Study of Long-term Safety and Efficacy of Myozyme in Patients With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored Enzyme Replacement Therapy (ERT) Studies [Phase 2]
An extension study assessing long-term safety and efficacy of Myozyme (alglucosidase alfa) in Pompe disease patients who were previously enr…
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CLINICAL_TRIALS trial_result · 2014-02-05Clinical Trial Update: Extension Study of Long-term Safety and Efficacy of Myozyme for a Single Patient With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored ERT Studies. [Phase 2]
This is an extension study evaluating long-term safety and efficacy of Myozyme (alglucosidase alfa) in a single patient with Pompe disease w…
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CLINICAL_TRIALS research · 2013-12-04Clinical Trial Update: A Long Term Follow up Study in Late-onset Pompe Disease
Clinical trial update detailing a long-term follow-up study in late-onset Pompe disease.
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CLINICAL_TRIALS research · 2013-08-30Clinical Trial Update: A Natural History Study of Adult Onset Pompe Disease Using Muscle MRI
A natural history study of adult-onset Pompe disease using muscle MRI; the trial status is currently unknown.
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CLINICAL_TRIALS trial_result · 2013-01-01Clinical Trial Update: Newborn Screening Assay of Pompe's Disease [NA]
The document reports the completion of a clinical trial evaluating a newborn screening assay for Pompe disease conducted by National Taiwan …
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FDA approval · 2012-07-16FDA Approved: LUMIZYME — Supplemental Application (Genzyme Corporation)
FDA approved a supplemental application for Lumizyme (alglucosidase alfa) submitted by Genzyme Corporation.