Paving a way to treat spastic paraplegia 50

Spastic paraplegia 50 (SPG50) is a rare neurodegenerative disease caused by loss-of-function mutations in AP4M1. There are no effective treatments for SPG50 or any other type of SPG, and current treatments are limited to symptomatic management. In this issue of the JCI, Chen et al. provide promising...

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Bibliographic Details
Main Authors: Jonathan R. Brent, Han-Xiang Deng
Format: Article
Language:English
Published: American Society for Clinical Investigation 2023-05-01
Series:The Journal of Clinical Investigation
Online Access:https://doi.org/10.1172/JCI170226