Pompe Disease
📧 Sign up to get email alerts when something new happens for Pompe Disease.
Sign up freeTop drugs studied for this disease
-
FDA approval · 2012-07-09FDA Approved: LUMIZYME — Supplemental Application (Genzyme Corporation)
FDA approved a supplemental application for Lumizyme (alglucosidase alfa) from Genzyme Corporation.
-
CLINICAL_TRIALS trial_result · 2012-03-16Clinical Trial Update: 3D Echocardiography Managing Infantile Pompe's Disease
An update indicates that the clinical trial evaluating 3D echocardiography for managing infantile Pompe disease has been terminated.
-
CLINICAL_TRIALS trial_result · 2012-01-05Clinical Trial Update: Late-Onset Treatment Study Extension Protocol [Phase 4]
Phase 4 study extension for late-onset Pompe disease and glycogen storage disease type II; the trial is completed and results were posted in…
-
CLINICAL_TRIALS research · 2011-12-08Clinical Trial Update: Pompe Prevalence Study in Patients With Muscle Weakness Without Diagnosis [NA]
ClinicalTrials.gov update notes a completed prevalence study investigating Pompe disease in patients with unexplained muscle weakness.
-
CLINICAL_TRIALS research · 2011-12-01Clinical Trial Update: Detection of Pompe Disease in Adult Patients With Myopathies of Uncertain Origin or With Asymptomatic Hyper-CK-emia
The document describes a clinical trial investigating detection of Pompe disease in adults with myopathies of uncertain origin or with asymp…
- Want full summaries, importance scores, and drug timelines? Upgrade to Plus or Pro.
-
FDA approval · 2011-09-12FDA Approved: LUMIZYME — Supplemental Application (Genzyme Corporation)
FDA approved a supplemental application for Lumizyme (alglucosidase alfa) from Genzyme Corporation.
-
CLINICAL_TRIALS research · 2011-08-04Clinical Trial Update: Screening for Early Detection and Prevention of Pompe Disease in Israel Using Tandem Mass Spectrometry
A clinical trial update describing screening for early detection and prevention of Pompe disease in Israel using tandem mass spectrometry.
-
CLINICAL_TRIALS trial_result · 2011-04-22Clinical Trial Update: High Dose or High Dose Frequency Study of Alglucosidase Alfa [Phase 4]
Phase 4 clinical trial evaluating high-dose or high-dose-frequency alglucosidase alfa in Pompe disease completed with results posted in 2011…
-
CLINICAL_TRIALS trial_result · 2010-07-23Clinical Trial Update: A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease [Phase 3]
A Phase 3, placebo-controlled trial evaluating the safety and efficacy of Myozyme (alglucosidase alfa) in late-onset Pompe disease has compl…
-
FDA approval · 2010-05-24FDA Approved: LUMIZYME — Original Application (Genzyme Corporation)
FDA approved Lumizyme (alglucosidase alfa) through its original application by Genzyme for Pompe disease, establishing enzyme replacement th…
-
EMA safety_alert · 2009-01-16Priority access for children during Myozyme supply shortage
EMA announces priority access for children during the supply shortage of Myozyme (alglucosidase alfa), to ensure treatment continuity for pe…
-
CLINICAL_TRIALS research · 2008-06-05Clinical Trial Update: A Study to Evaluate the Effects of Pharmacological Chaperones in Cells From Patients With Pompe Disease
A completed study evaluated the effects of pharmacological chaperones on cells derived from patients with Pompe disease.