News Voice Quality of Patients with Late-Onset Pompe Disease Deteriorates Over Time, Study Shows

News Audentes Therapeutics Plans to File Investigational New Drug Application for Potential Pompe Disease Gene Therapy This Year

News First Late-Onset Pompe Disease Patient Treated With AT-GAA in Phase 3 Trial

News Rising Healthcare Costs Strain EU Budgets Even as New Therapies Flourish

News Late-Onset Pompe Disease Mix with Findings of Amyloidosis in Patient, Case Study Reports

News Enzyme Replacement Therapy for Pompe Disease Could Be Derived From Rice, Study Suggests

News Early, Higher Myozyme Dose Effective in Boy With Classic Infantile-onset Pompe, Study Reports

News Breathing Exercises Enhanced Respiratory Capacity of Late-onset Pompe Disease Patients

News Most Pompe Disease Patients Should Practice Pulmonary Hygiene on Daily Basis, Study Suggests

News Low Activity of Enzymes Involved in Pompe, Fabry Diseases Linked to More Dilated Arteries in Brain, Study Reports

News Break in ERT Treatment Linked to Worse Outcomes in Late-Onset Pompe Patients, Study Reports

News Lantern Project Intends to Streamline Diagnosis of Pompe, Other Rare Diseases

News Methotrexate Seen to Counter Immune Reaction to Enzyme Replacement Therapy in Small Study

News Advocacy Groups, Doctors Question Rising Prices of Rare Disease Treatments

News Spark Therapeutics’ SPK-3006 Shows Promising Preclinical Data for Treating Pompe Disease

News #NORDSummit – More Than 700 Expected to Attend Oct. 15-16 Rare Disease Summit in Washington

News Screening Programs May Identify Newborns at Risk for Late-onset Pompe Disease, Other Lysosomal Storage Disorders

News Amicus Announces Plans for Developing AT-GAA to Treat Pompe Disease

News Newborn Screening Better Than Clinical Exam at Detecting Pompe Disease, Study Shows

News Perlara Partners with Notre Dame to Develop Therapies for Pompe, Cori Diseases

News Clenbuterol Seen to Improve ERT Effectiveness in Pompe Disease Clinical Trial

News FDA Clears Diagnostic Kit to Screen Newborns for Pompe and 5 Other Lysosomal Storage Disorders

News Immunomodulation Plus Myozyme May Help Infants with Pompe Disease, But Seen to Carry Risks in Study

News Quantitative Muscle MRI Can Help Monitor Late-onset Pompe Disease, Study Shows

News Higher Lumizyme Dose May Help Prevent Pompe Progression in Infants, Case Report Suggests

News Severe Heart Disease Rare in Patients with Late-onset Pompe Disease, Study Suggests

News Gene Therapy Using Improved Virus Holds Promise for Pompe Disease Treatment, Mouse Study Shows

News Infantile-onset Pompe Disease Cases Highlight Importance of Early Diagnosis, Treatment

Just Published, News Neuron-specific Gene Therapy May Improve Motor Deficits in Pompe Disease, Mouse Study Shows

News US ‘Right to Try’ Law Meets with Mix of Praise and Criticism, Including Among Those with Rare Diseases

News Pompe Patients Have Abnormalities in Artery that Supplies Blood to the Brain, Study Shows

News #ERDC2018 – When Treating Rare Disease Patients, Don’t Overlook Quality of Life, Panel Urges

News NIH Awards $325K Small Business Grant to Develop Risk Assessment Tool for Pompe Disease

News ‘Rare Impact Awards’ Dinner Marks Orphan Drug Act and NORD at 35

News #ECRD2018 – EU Must Do More for Rare Disease Patients, Eurordis Leaders Say

News ERT Fails to Prevent Long-Term Effects of Infantile Pompe Disease to Brain, Study Shows

News Amicus Recruiting Pompe Patients for Phase 1/2 Trial of AT-GAA Combo Therapy

News Researchers Develop Cheap, More Sensitive Screening Test for Pompe Diagnosis, Study Reports

News Amicus Therapeutics Raising Awareness in April About Pompe Disease, Fabry Disease

News #AAN2018 — Investigational Pompe Therapy ATB200/AT2221 Shows Promising Results in Phase 1/2 Trial

News Duke Researchers Initiating Phase 1 Trial on New Gene Therapy for Pompe Disease

News Shire, NanoMedSyn Team Up to Research Potential Therapy for Pompe Disease

News Lumizyme Found Effective in Pompe Disease Patients Previously Taking Myozyme, Trial Shows

News CHOP Researchers Restore Cellular Energy Signals, Offering Possibility of Treating Mitochondrial Diseases