News of interest about Pompe disease
Live videoconference (in Spanish) organized by the Spanish Pompe Patients Association (AEEPOMPE), presented by Dr. Dominguez Gonzalez, Neurology Department (Neuromuscular Unit), Hospital 12 de Octubre, Madrid. July 7, 4:00 PM CEST. Open access through the link.
July 2026
Researchers present promising results from phase I/II gene therapy trials for late-onset Pompe disease.
Several Spanish autonomous communities incorporate Pompe disease into their newborn screening program.
Health Policy • November 2025The European Society for Neuromuscular Diseases publishes an update of its recommendations for monitoring Pompe patients.
Clinical Guidelines
October 2025
Summary of activities organized by patient associations worldwide on the occasion of International Pompe Day.
Community
April 2025
A European multicenter study evaluates the impact of ERT on quality of life in patients with late-onset Pompe disease.
Research
September 2025
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