Nature

Diagnostic challenges for Pompe disease: An under-recognized cause of floppy baby syndrome

Floppy baby syndrome is a condition associated with impaired development of motor skills and signs of hypotonia, profound weakness, and rag-doll features. 1,2 Because numerous disorders can give rise ...
Monthly Prescribing Reference

Pombiliti Plus Opfolda Approved for Late-Onset Pompe Disease

Credit: Getty Images. Pombiliti must be administered in combination with Opfolda. The Food and Drug Administration (FDA) has approved Pombiliti ™ (cipaglucosidase alfa-atga) in combination with ...
Nature

Ambulatory electrocardiogram analysis in infants treated with recombinant human acid α-glucosidase enzyme replacement therapy for Pompe disease

Purpose: Infantile Pompe disease is caused by deficiency of lysosomal acid α-glucosidase. Trials with recombinant human acid α-glucosidase enzyme replacement therapy (ERT) show a decrease in left ...
News Medical

Spark Therapeutics launches gene therapy clinical trial for late-onset Pompe disease

Pompe disease is a hereditary genetic disorder caused by a deficiency of acid alpha-glucosidase leading to build-up of glycogen in the lyosomes, which then causes cell damage in various tissues, in ...
Nasdaq

Amicus Therapeutics Publishes Positive Post-Hoc Analysis of Cipaglucosidase Alfa-Atga + Miglustat in Late-Onset Pompe Disease

Amicus Therapeutics has released a post-hoc analysis from the PROPEL study, published in Muscle and Nerve, which evaluated the efficacy of cipaglucosidase alfa-atga combined with miglustat (cipa+mig) ...
Business Wire

Pompe Disease - A Pipeline Analysis Report | Technavio

LONDON--(BUSINESS WIRE)--Technavio has announced its latest pipeline analysis report on the Pompe disease market. The report comprises an in-depth analysis of the pipeline molecules under ...
Mena FN

Pompe Disease Market Analysis Report 2025-2035: Enzyme Replacement Therapies And Approvals Of Myozyme And Nexviazyme Drive Steady Growth

(MENAFN- GlobeNewsWire - Nasdaq) Pompe disease is a rare genetic disorder resulting from GAA gene mutations, leading to acid alpha-glucosidase deficiency and glycogen buildup. Key symptoms include ...