They Carry a Gene for ALS but Aren’t Sick. What Does Medical Research Owe Them?

By Jason Mast
September 21, 2023
Pictured left to right: Matthew B. Harms, MD and Neil A. Shneider, MD, PhD

Pictured left to right: Matthew B. Harms, MD and Neil A. Shneider, MD, PhD

“You want to be cautious,” said Matthew Harms, who runs the ALS Families Project at Columbia, adding that he agrees there needs to be more advocacy and research for carriers.

Neil Shneider, a task force member and neurologist specializing in genetic ALS at Columbia responded that, “It really is about risk.” New gene-targeted drugs are not benign, Shneider pointed out. They rely on short strands of DNA, called antisense, that have caused brain swelling in other trials. [read more]

Source: STAT News
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