Amicus Therapeutics Inc.

Research in late-onset Pompe disease includes adult subjects and reflects a focus on long-term treatment use in lysosomal storage disorder care. The sponsor studies ATB200 with AT2221 and cipaglucosidase alfa with miglustat in relation to safety and treatment performance.

• ATB200/AT2221 co-administration
• Cipaglucosidase alfa and miglustat
• Adult Pompe disease
• Tolerability in chronic therapy

This area includes attention to both biochemical support strategies and clinical use in adults living with Pompe disease.

Paediatric research covers infantile-onset Pompe disease from infancy through adolescence, including both enzyme replacement therapy-experienced and ERT-naïve children. The sponsor evaluates cipaglucosidase alfa/miglustat as a treatment approach in very young patients with Pompe disease.

• Infantile-onset Pompe disease
• Paediatric treatment
• ERT-experienced children
• ERT-naïve children

Clinical interest spans early childhood to late adolescence, with emphasis on treatment use in a rare neuromuscular metabolic condition.

The sponsor’s portfolio includes paediatric studies across lysosomal storage disorders, especially Fabry disease and Pompe disease. These trials address treatment considerations in younger patients where growth, developmental stage, and organ involvement may influence care.

• Children with Fabry disease
• Children with Pompe disease
• Age-specific treatment use
• Rare metabolic disease

Research activity in this area reflects sustained attention to therapies for inherited disorders affecting enzyme function and cellular metabolism.