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Early detection and treatment of Pompe disease give patients hope
Infantile and late-onset Pompe disease are the two subtypes. The entire or almost total lack of GAA results in the infantile variant. During the first several months of life, symptoms appear.
pharmaphorum1y
Sanofi finds new Pompe disease therapy in Maze
Sanofi is already a big player in the Pompe disease market with injectable drugs like Nexviazyme and Myozyme/Lumizyme, but has now set its sights on an oral drug candidate from Maze Therapeutics.
pharmaphorum2y
CHMP backs Amicus' therapy for Pompe disease
Amicus Therapeutics could be just weeks away from getting EU approval for its new Pompe disease therapy Pombiliti after getting a positive opinion on the drug from the EMA's human medicines committee.
precisionmedicineonline5d
FDA Clears Aro Biotherapeutics' siRNA Drug IND for Late-Onset Pompe Disease
The company has already kicked off an open-label Phase I trial of ABX1100 in Canada following positive results from a study of healthy volunteers.
Enzyme Replacement Therapy Market Size, Growth & Forecast By 2033
The global enzyme replacement therapy market size was valued at USD 10.12 billion in 2024 and is projected to grow from USD 11.35 billion in 2025 to reach USD 21.29 billion by 2033, exhibiting a CAGR ...