Pompe Disease
📧 Sign up to get email alerts when something new happens for Pompe Disease.
Sign up freeTop drugs studied for this disease
-
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…
-
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…
-
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…
-
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.
-
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.
- Want full summaries, importance scores, and drug timelines? Upgrade to Plus or Pro.
-
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.
-
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.
-
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…
-
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.
-
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…
-
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 …
-
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…
-
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…
-
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…
-
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; …
-
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…
-
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…
-
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.
-
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…
-
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…
-
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…
-
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…
-
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…
-
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…
-
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…