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Length of experience

Myozyme® was approved in the EU in 20061

Patient groups

Myozyme® can change the natural history of Pompe disease, for patients regardless of age, gender or symptoms1,3

Early treatment initiation

Early initiation of treatment has been linked to a greater benefit and better clinical outcomes in IOPD and LOPD3,4


Myozyme® is indicated for long-term enzyme replacement therapy (ERT) in patients with a confirmed diagnosis of Pompe disease (acid alpha glucosidase deficiency).1

Myozyme® stabilises pulmonary function in patients with LOPD5

Patients treated with Myozyme® maintained improvements from baseline in walking distance, measured by the 6MWT, for up to 104 weeks vs. placebo5,6

Early initiation of treatment helps improve patients’ walking distance from baseline, potentially maximising their independence vs. placebo5,6

ERT results in significant improvements in survival of late-onset Pompe disease patients vs. those not treated with ERT*2

Observational data found ERT to be associated with a mortality hazard ratio of 0.41 relative to patients not receiving ERT, which is a 59% reduction in the likelihood of death. 28 patients died in the non-ERT group and 18 died in the ERT group.9 This extrapolates to approximately 1 extra year of life for every 8 years of ERT.2 ERT has also been shown to reduce mortality in infantile-onset Pompe disease compared to those not treated with ERT.7

*International observational study including data from 283 adult patients with Pompe disease2.

ERT has a positive impact on quality of life in adult patients with Pompe disease compared to baseline8.

An international survey* showed that a significant decrease in the physical component summary score of the quality-of-life measure, SF-36, in Pompe disease patients (-0.73 score points per year [sp/y]) before starting ERT8.

*International survey of 174 patients, assessed for quality-of-life using the SF-36 instrument, annually between 2002 and 2012; median follow-up time: 4 years (range 0.5-8)8

Long-term benefits of MYOZYME treatment in LOPD

Study design9

A nationwide, prospective study analysed the long-term effects of enzyme replacement therapy (ERT) in 102 Dutch adult patients, ranging in age from 24 to 76 years, with Pompe disease who had not yet received ERT before enrollment on January 1, 2005. If treated, patients received Myozyme® at a dosage of 20 mg/kg every 2 weeks. Pulmonologic data and muscle function were assessed among other variables.

Clinical assessments took place every 3 to 6 months before and after the start of Myozyme; the median overall follow-up duration was 6.1 years and was compared against the natural history course extrapolated data.

Distance walked in 6MWT over the course of disease after the start of ERT9

Maximizing independence in the long term9 reduces risk of wheelchair dependence10

Myozyme® can help keep long-term independence possible for adult patients with LOPD9

Individual patient response to ERT treatment on the Rasch-built Pompe-specific activity scale (R-PAct)12

Myozyme® can help keep long-term independence possible for adult patients with LOPD9

In patients with IOPD, Early initiation of Myozyme® improves clinical outcomes in maintaining motor function and prolonging survival versus historical, untreated controls1,11

Kaplan-Meier estimate of time from birth to invasive ventilator use or death11

  1. Myozyme Summary of Product Characteristics. Accessed October 2022.
  2. Güngör D, et al. Orphanet J Rare Dis. 2013;8:49.
  3. Kishnani PS, et al. Genet Med. 2006; 8(5):267–288.
  4. Barba-Romero MA, et al. Rev. Neurol. 2012;54(8):497–507.
  5. van der Ploeg AT, et al. N Engl J Med. 2010;362(15):1396-1406.
  6. van der Ploeg AT, et al. Mol Genet Metab. 2012;107(3):456-461.
  7. Chen M, et al. Cochrane Database Syst Rev. 2017;11:CD011539.
  8. Güngör D, et al. J Inherit Metab Dis. 2016;39(2):253–260.
  9. Kuperus E, et al. Neurology. 2017;89(23):2365–2373.
  10. van der Meijden JC, et al. Orphanet J Rare Dis. 2018;13(1):82.
  11. Kishnani PS, et al. Neurology. 2007;68(2):99-109.

MAT-XU-2302935 (v1.0) Date of preparation: October 2023