Cytokinetics announced that it is stopping COURAGE-ALS, a Phase 3 trial investigating oral reldesemtiv in treating people with amyotrophic lateral sclerosis (ALS), due to a lack of efficacy compared with a placebo. In a review of unblinded interim trial findings at a second planned data monitoring committee meeting,…
A Phase 3b study evaluating the long-term safety and effectiveness of two dosing regimens of Radicava ORS (edaravone) — an approved oral formulation of the medicine — in people with amyotrophic lateral sclerosis (ALS) is now fully enrolled. The study was designed to fulfill a post-marketing commitment that…
The other day my husband, Todd, told me he forgot to ask the caregiver to spray out his clicker tube for his HeadMouse. Because he’s paralyzed from ALS, he clicks his computer mouse by sipping and puffing on a straw, and we only need to replace it once…
The U.S. Food and Drug Administration (FDA) will hold an advisory committee meeting to discuss BrainStorm Cell Therapeutics‘ application to approve NurOwn for amyotrophic lateral sclerosis (ALS). Such a meeting, called an ADCOM, is normally for when a therapy is under regulatory review so experts can discuss its…
Difficulties in measuring the progression of amyotrophic lateral sclerosis (ALS) among patients with advanced disease can cause complications in clinical trials, according to a new analysis that highlights how such problems may have affected the failed NurOwn study. The work was led by scientists at BrainStorm…
In most cases of amyotrophic lateral sclerosis (ALS), dysfunction of a protein known as TDP-43 leads to abnormally low levels of another protein, stathmin-2, which is sufficient to drive nerve cell dysfunction, new data suggest. In an aim to restore stathmin-2 levels, researchers created a designer DNA treatment. Now,…
“So tell us, Dagmar, what’s your latest project?” The question came from my neurologist as we chatted during my recent ALS clinic visit. She asked not only for herself, but for the medical student who was also in the room. As I prattled on, part of my brain reflected…
On average, a person with amyotrophic lateral sclerosis (ALS) will require a wheelchair to get around within two years of the initial onset of disease symptoms. Findings underscore the condition’s rapid advance, and the need for new ALS treatments that work to slow disease progression, its researchers noted.
On a bitterly cold morning in New York last weekend, I woke before the sun and walked through the empty city streets to the Times Square subway station. From there, I caught a train to Brooklyn, the starting line for the United Airlines NYC Half, where more than…
Changes in neurofilament light chain (NfL) levels — a biomarker of nerve cell damage — were deemed by an advisory committee reasonably likely to predict clinical efficacy from tofersen in people with amyotrophic lateral sclerosis (ALS) caused by SOD1 gene mutations. The unanimous vote from the Peripheral and Central Nervous…
