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News about "Late-onset Pompe disease"
Sanofi's Nexviazyme Meets Primary Goal in Phase 3 Study for Infantile-Onset Pompe Disease
Sanofi's Phase 3 Baby-COMET study showed Nexviazyme improved ventilator-free survival in infants with infantile-onset Pompe disease, supporting plans for a US regulatory filing to expand the therapy's approved indication.
Late-Onset Pompe Disease | 01/07/2026 | By News Bureau
AskBio Doses First Participant in Phase 1/2 Trial for Pompe Disease Gene Therapy
AskBio has dosed the first participant in its Phase 1/2 PROGRESS-GT LOPD trial evaluating investigational gene therapy AB-1009 for late-onset Pompe disease, advancing research into potential long-term treatment solutions for the rare genetic disorder.
Late-onset Pompe Disease | 14/05/2026 | By News Bureau
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