Health AffairsOpinion

Refining FDA’s Plausible Mechanism Framework, Part 1: Understanding The Draft Guidance And Clarifying The Framework’s Scope

This is part one of a two-part article offering proposals to improve the FDA’s guidance to implement a new approval framework ...
Nature

Lysinuric Protein Intolerance and Associated Pathophysiological Mechanisms

Lysinuric protein intolerance (LPI) is an autosomal recessive metabolic disorder caused by pathogenic variants in the SLC7A7 gene, which encodes the y+LAT1 subunit of the system y+L cationic ...
Nasdaq

CAMP4 Therapeutics Reports Positive Results in SYNGAP1-Related Disorders and Urea Cycle Disorders at ASGCT Annual Meeting

CAMP4 Therapeutics reports promising results for CMP-SYNGAP-01 and CMP-CPS-001 in treating genetic disorders, with favorable safety data. CAMP4 Therapeutics announced promising results from its ...