Ulefnersen shows favorable safety profile, may be efficacious for rare type of ALS

Neil Shneider, MD, PhD, physician, scientist, and director of the Eleanor and Lou Gehrig ALS Center at Columbia University, talks about how ulefnersen, an experimental drug, that showed promising results in benefitting some patients with a rare form of Amyotrophic Lateral Sclerosis (ALS) and how this may help with researching treatment for other variants of ALS. 

“There are clinical and pathological similarities between genetic and more common forms of ALS, which gives us real reasons to think they have a common underlying biology,” said Dr. Shneider. “By comparing these ultra-rare patients and their response to individualized therapy, we hope to learn something about the biology of the disease that will eventually help a larger percentage of ALS patients.” [read more]

Source: Healio