biotech
Advances in gene therapy for Pompe
Researchers present promising results from phase I/II gene therapy trials for late-onset Pompe disease.
science
Research
December 2025
News of interest about Pompe disease
Researchers present promising results from phase I/II gene therapy trials for late-onset Pompe disease.
December 2025
Several Spanish autonomous communities incorporate Pompe disease into their newborn screening program.
The European Society for Neuromuscular Diseases publishes an update of its recommendations for monitoring Pompe patients.
Clinical Guidelines • October 2025Summary 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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