
A simple patient survey revolutionized ALS drug development in the 1990s. Its creator says it may be time for an upgrade
It’s been another tough year for ALS therapies. New trials that sparked patient hopes either fell short of statistical significance or failed to provide enough evidence for the FDA to recommend moving forward without another look. The end result is largely more of the same for patients — little progress in a fatal disease with only two approved treatments.
That frustrating slog has stirred passionate debate within the patient and advocacy communities, largely around things like trial design, compassionate use programs or even what the bar for approval should be. Long overlooked in those discussions, however, is a tool used to evaluate every ALS therapy that’s drawing increasing scrutiny.
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