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Big data for a rare disease: FNIH takes on ALS

ARTICLE | Discovery & Translation

In partnership with FDA, industry and disease foundations, FNIH is creating a one-stop shop for data on the rare disease to spur diagnostics and therapeutics development 

By Selina Koch, Executive Editor

May 21, 2024 9:06 PM UTC

FNIH is stepping in to push forward the search for effective treatments for ALS, a rare disease that has both caught the public spotlight and confounded drug development, with a roller coaster of expectations and disappointments raising the stakes to find solutions for patients with the devastating disease. 

Five months into the year, 2024 has already brought seven clinical failures of therapies for amyotrophic lateral sclerosis (ALS), including the high-profile Phase III miss of Relyvrio from Amylyx Pharmaceuticals Inc. (NASDAQ:AMLX) leading to removal of the drug — one of very few to receive an FDA approval for the indication — from the U.S. market. …