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Update on Pompe Disease Treatment.

Sanofi-Genzyme’s newest ERT awarded Early Access to Medicine Status

Avalglucosidase-alfa was awarded EAMS (Early Access to Medicines Status) in February 2021.

The treatment was designated a Promising Innovative Medicine in September 2020. Click on link for more information about PIM.

Link to PIM Information

This has now been followed with EAMS designation. This means that, although it remains an unlicensed medicine, patients who meet the qualifying criteria can approach their treating physician to ask about whether it is right for them.

Link to UK Government information


AGSD-UK welcomes this progress and will continue to update our Pompe membership on further developments.