Correspondence on “Stability of alglucosidase alfa in 0.9% sodium chloride for enzyme replacement therapy in patients with Pompe disease: insights from enzyme activity and cellular uptake measurements” by Barzel et al

The study by Barzel et al provides timely and clinically relevant evidence that ready-to-administer alglucosidase alfa retains enzyme activity and cellular uptake capacity for up to 11 days under refrigerated or frozen conditions,1 although whether such stability persists under real-world preparation and transport conditions remains to be established and is therefore worth discussing alongside other implications and caveats. This finding, if confirmed and extended, carries substantial implications for patients, clinicians and pharmacists engaged in the long-term management of Pompe disease.