Complicaciones y seguimiento de la enfermedad de Pompe

Diaz-Manera J, Kishnani PS, Kushlaf H, Ladha S, Mozaffar T et al.; COMET Investigator Group.

Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial.

Lancet Neurol. 2021 ;20:1012-1026

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Schoser B, Roberts M, Byrne BJ, Sitaraman S, Jiang H et al;; PROPEL Study Group.

Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial.

Lancet Neurol. 2021 ;20:1027-1037

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Harlaar L, Hogrel JY, Perniconi B, Kruijshaar ME, Rizopoulos D et al..

Large variation in effects during 10 years of enzyme therapy in adults with Pompe disease.

Neurology. 2019;93:e1756-e1767

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Nuñez-Peralta C, Alonso-Pérez J, Llauger J, Segovia S, Montesinos P et al..

Follow-up of late-onset Pompe disease patients with muscle magnetic resonance imaging reveals increase in fat replacement in skeletal muscles.

J Cachexia Sarcopenia Muscle. 2020 ;11:1032-1046

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