Compositions and methods for treating X-linked myotubular myopathy

JP2024510972A5Inactive Publication Date: 2025-03-19ASTELLAS GENE THERAPIES INC
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Patent Information

Application Number
JP2023555441
Authority / Receiving Office
JP · JP
Patent Type
Applications
Current Assignee / Owner
Priority Date
2021-03-11
Filing Date
2022-03-11
Publication Date
2025-03-19
Estimated Expiration
Not applicable · inactive patent

AI Technical Summary

Technical Problem

There is no effective method for weaning chronically ventilated patients with X-linked myotubular myopathy (XLMTM) from mechanical ventilation, as they require continuous respiratory support due to severe muscle weakness and respiratory failure.

Method used

A method for assessing a patient's readiness to initiate or continue weaning from a ventilator, involving specific physiological parameters such as maximum inspiratory and expiratory pressures, oxygen saturation, carbon dioxide levels, serum bicarbonate levels, and motor function scores, followed by administration of a viral vector encoding MTM1 to enhance muscle function.

Benefits of technology

Enables safe weaning of patients from mechanical ventilation, potentially reducing the need for ventilator support and improving quality of life by enhancing MTM1 expression and muscle function.

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Abstract

The present invention provides compositions and methods for treating neuromuscular disorders. In certain embodiments, the present invention provides compositions and methods for assessing readiness and continuation of weaning from mechanical ventilation in subjects suffering from X-linked myotubular myopathy (XLMTM).
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