New agent approved for late-onset Pompe disease
FDA has approved alglucosidase for patients aged 8 years and older for the treatment of late-onset Pompe disease.
FDA has approved alglucosidase alfa (Lumizyme, Genzyme) for patients aged 8 years and older for the treatment of late-onset (noninfantile) Pompe disease.
The agent was approved with a risk evaluation and mitigation strategy and will be available only through a restricted distribution system. The product labeling will also contain a boxed warning regarding an increased risk of anaphylaxis and severe allergic and immune-mediated reactions.
The approval of this agent was based on a clinical study in 90 patients aged 10 to 70 years with late-onset Pompe disease. In this study, the most common adverse events included infusion-related reactions.
Comparison of teen- and parent-reported estimates of social and emotional support, 2021 to 2022
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