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Table 4 Recommendations for initiation of alglucosidase alfa ERT based on patient status

From: Diagnosis and treatment of late-onset Pompe disease in the Middle East and North Africa region: consensus recommendations from an expert group

Patient symptomatology

Recommendations for alglucosidase alfa ERT

Presymptomatic with no signs or symptoms

ERT not necessary. Patient should be monitored every 6 months and ERT initiated if there is evidence of clinical deterioration

Presymptomatic with abnormal muscle imaging or abnormal muscle biopsy

ERT should be considered on a case-by-case basis

Symptomatic with signs or symptoms

ERT should be initiated in patients with muscle weakness or reduced pulmonary function

Patients with markedly advanced disease, who have lost ambulation and are ventilation-dependent

ERT should be administered for 1 year with evaluation of effectiveness

  1. Adapted with permission from Cupler et al. Consensus treatment recommendations for late-onset Pompe disease. Muscle Nerve. 2012;45(3):319–33. [6] © 2011 Wiley Periodicals, Inc
  2. ERT enzyme replacement therapy