US EditionExperimental ALS Drug Shows Unprecedented Recovery in some Patients
- Researchers at Columbia University announced on May 23, 2025, that an experimental drug called ulefnersen showed unprecedented recovery in some ALS patients.
- The drug targets a rare, aggressive form of ALS caused by mutations in the FUS gene that affect adolescents and young adults, representing 1-2% of ALS cases.
- Shneider developed ulefnersen, an antisense oligonucleotide therapy aimed at lowering toxic FUS protein levels, and worldwide use in expanded access programs has involved more than two dozen patients, with no serious adverse effects reported.
- In a case series of 12 patients published in The Lancet, one young woman regained the ability to walk and breathe unaided, and a man treated before symptoms emerged has shown no disease development after three years.
- These results suggest early intervention may slow or reverse ALS progression, motivating ongoing global trials and hopes for ulefnersen's approval as a precision medicine therapy.
16 Articles
16 Articles
Experimental drug may benefit some patients with rare form of ALS
When Columbia neurologist and scientist Neil Shneider speaks to his ALS patients who volunteer for experimental therapies, he's unwaveringly honest. "Patients always ask me, 'What can I hope to get out of this?"' Shneider says. "And I always say, in most clinical trials, our hope is that we can slow the disease or maybe even halt progression."
Experimental drug may benefit some patients with rare form of ALS - Scientific Inquirer
When Columbia neurologist and scientist Neil Shneider speaks to his ALS patients who volunteer for experimental therapies, he’s unwaveringly honest. “Patients always ask me, “What can I hope to get out of this?” Shneider says. “And I always say, in most clinical trials, our hope is that we can slow the disease or maybe even halt progression.” So it was a big surprise when some of the patients treated with an experimental drug—a therapy that eme…
A young woman who could not walk or breathe on her own because of a rare genetic form of amyotrophic lateral sclerosis (ELA-FUS) has regained her functions after receiving an experimental treatment called ulefnersen. The breakthrough, led by researchers from Columbia University and published in ‘The Lancet’, has been tested in 25 other patients, 12 of whom are part of the study; in two of them the results are hopeful and without presences.
A young woman who could not walk or breathe alone due to a rare genetic form of amyotropic lateral sclerosis (ELA-FUS) has regained her functions after receiving an experimental treatment called ulefnersen. Researchers at the Irving Medical Center at Columbia University (United States) have presented data on 12 patients, all treated with a new therapy for a rare form of amyotropic lateral sclerosis (ELA) caused by a genetic mutation in a gene ca…
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