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Biologics in Therapy

, 1:1

First Online: 12 August 2011Received: 14 March 2011

Abstract

IntroductionPompe’s disease is a metabolic myopathy caused by a deficiency of the enzyme alpha-glucosidase. Patients with late-onset Pompe’s disease have progressive muscle weakness, which also affects pulmonary function. Since the advent of specific treatment for Pompe’s disease, enzyme replacement therapy with alpha-glucosidase, the prognosis of the disease has changed.

MethodsWe report the case of the first patient treated in Spain with home therapy, and the effects on her clinical status of a reduction in treatment frequency.

ResultsA worsening was seen in the patient’s neuromuscular assessment on different scales, after two discontinuations during the patient’s usual administration frequency.

ConclusionIt is essential to keep an adequate administration schedule to maintain the clinical benefits of enzyme therapy.

Keywordsalpha-glucosidase replacement enzyme maltase acid deficiency neuromuscular disease Pompe’s disease To view enhanced content go to www.biologicstherapy-open.com

This article is published with open access at Springerlink.com

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Autor: Emilia Barrot Cortés - Juana María Barrera Chacón

Fuente: https://link.springer.com/







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