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late-onset Pompe disease

Pharma & Biotech

Oral Pompe therapy S-606001 moves to Phase 2 as Shionogi expands rare disease focus

Shionogi's S-606001 enters global Phase 2 as the first oral GYS1 inhibitor tested as an add-on to ERT in late-onset Pompe disease. Read the analysis.

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Pharma & Biotech

Can AB-1009 reshape treatment for late-onset Pompe? AskBio prepares for first patient in U.S.

AskBio’s AB-1009 gene therapy for late-onset Pompe secures FDA IND clearance. Find out what this milestone means for AAV innovation and patient outlook.

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