MUSCLE SPECIFIC RECEPTOR KINASE AND MODULATION THEREOF

摘要

Agents, compositions, and medicaments that pertain to modulating the activity of muscle specific kinase receptor (MuSK) and methods and uses thereof to modulate MuSK activity are encompassed herein. Also encompassed are screening assays to identify modulators of MuSK activity. Agents identified using the screening assays described herein are envisioned for use as therapeutics, alone or in compositions or in medicaments, to alleviate or delay motor dysfunction in subjects afflicted with a disorder associated with nerve terminal loss or fragmentation, such as amyotrophic lateral sclerosis, sarcopenia, or anti-MuSK myasthenia gravis.

主权项

1. A method for delaying motor dysfunction in a subject, the method comprising: administering an agent that increases muscle specific receptor kinase (MuSK) activity to the subject in a therapeutically effective amount sufficient to increase MuSK activity in the subject and thereby improve motor function in the subject.