Stabilisation of motor function two years after switching to alfa-sialidase in Pompe disease

A French study analysed real-world data from 47 adults with late-onset Pompe disease who switched to alfa-glucosidase (Nexviadyme) after treatment failure with alfa-glucosidase (Myozyme).

• Motor decline stabilised: results on the six-minute walk test were deteriorating prior to the change in treatment, then improved during the first year, before stabilising in the second year.
• Responses varied within the cohort, however: 20 patients showed significant improvement, whilst three ‘poor responders’ experienced a marked worsening.
• No significant changes in respiratory function were observed (a trend towards slower deterioration).

Longer-term follow-up will be necessary to draw conclusions regarding larger cohorts and, potentially, to understand the prognostic factors for response