Compound for treating spinocerebellar ataxia type 3

JP2025521545A5Pending Publication Date: 2026-06-30PTC THERAPEUTICS INC
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Patent Information

Authority / Receiving Office
JP · JP
Patent Type
Applications
Current Assignee / Owner
PTC THERAPEUTICS INC
Filing Date
2023-06-20
Publication Date
2026-06-30

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Abstract

One aspect of the present disclosure relates to compounds useful for improving pre-mRNA splicing in cells. In particular, another aspect of the present disclosure relates to substituted heteroaryl compounds, forms, and pharmaceutical compositions thereof, and methods of use for treating or alleviating spinocerebellar ataxia type 3 (SCA3), also known as Machado-Joseph disease (MJD).
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