Using the French Pompe disease registry, data from 29 patients treated with Nexviadyme after loss of efficacy with Myozyme were analyzed.
- After at least one year of treatment, the average deterioration in motor skills was reduced, while respiratory impairment did not progress.
- At individual level, there may be different responses to Nexviadyme: motor or respiratory improvement, or worsening despite treatment.
These real-life data provide a clearer picture of the therapeutic benefits of Nexviadyme, which has been granted early access in the event of Myozyme failure. Approval to market the product is currently being sought from the French health authority (Haute Autorité de Santé).
