A disease progression model comparing the long-term mobility and respiratory outcomes of adults with late-onset Pompe disease receiving cipaglucosidase alfa plus miglustat versus alglucosidase alfa.

J Comp Eff Res · 2026 · PMC13435892 · PMID 42454581

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nominally significantno p-value reported

Cipa + mig showed a nominally significant improvement in sitting %predicted forced vital capacity (%predicted FVC) compared with alg in the overall (combined naive and experienced) and ERT-experienced populations.

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