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January 16, 2019January 16, 2019

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

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Patients with late-onset Pompe disease demonstrated a deterioration in voice quality over a period of three years, but early treatment may stop this progressive impairment, a study reports. The ... Read more

January 9, 2019January 9, 2019

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

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Audentes Therapeutics plans to submit an investigational new drug (IND) application for its Pompe disease gene therapy AT845 this year. In a recent update, Audentes, a biotech company, discussed ... Read more

January 3, 2019January 3, 2019

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

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Amicus Therapeutics has dosed the first patient in the Phase 3 PROPEL clinical trial that aims to compare investigational therapy AT-GAA (ATB200/AT221) to standard treatment in patients with late-onset Pompe disease. ... Read more

December 20, 2018December 20, 2018

Rising Healthcare Costs Strain EU Budgets Even as New Therapies Flourish

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Cash-strapped governments across the 28-member European Union are struggling to control runaway healthcare expenditures — at exactly the same time as the promise of new but expensive therapies to ... Read more

December 19, 2018December 19, 2018

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

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Manifestations of Pompe disease — its suspected signs or symptoms — can mimic diagnostic findings of a rare type of amyloidosis, a case study reports. The report, “Pompe Disease ... Read more

December 12, 2018December 12, 2018

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

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A research team in Korea has developed a new method using rice to produce lab-made human acid alpha-glucosidase (GAA) for the treatment of Pompe disease. Their findings were published ... Read more

December 5, 2018December 5, 2018

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

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Early treatment with a four times higher dose of Myozyme (alglucosidase alpha) was safe and enabled normal motor development and normalization of markers of muscle damage and disease progression in ... Read more

November 28, 2018November 28, 2018

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

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Frequent inspiratory muscle training — a set of breathing exercises to strengthen the respiratory muscles making it easier to breathe — enhanced the breathing capacity of late-onset Pompe disease ... Read more

November 21, 2018November 21, 2018

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

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Most patients with juvenile- and adult-onset Pompe disease have difficulties coughing that put them at an increased risk for respiratory problems, but few of them incorporate techniques of pulmonary ... Read more

November 7, 2018November 7, 2018

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

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Lower activity of the two lysosomal enzymes underlying Pompe and Fabry diseases — acid alpha-glucosidase enzyme (GAA) and alpha-galactosidase A (GLA) — is linked to dilated arteries in the ... Read more

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