In amyotrophic lateral sclerosis (ALS), the loss of any one of four RNA-binding proteins turns off a gene called UNC13A that needs to stay active for nerve cells to communicate with each other — with three of them leading to an excess of another protein, REST — pointing to…
Loss of protein in ALS silences key nerve cell communication gene
“I still can’t understand you. Say it one more time.” My husband and I were having a simple conversation, and I was trying to emphasize my point of view, but my mouth and tongue simply wouldn’t coordinate. Instead of pausing to slow down, I just kept repeating the same garbled…
Full randomization is nearly complete in the Phase 2/3 COMBAT-ALS trial testing MN-166 (ibudilast) for the treatment of amyotrophic lateral sclerosis (ALS), its developer, Medicinova, reported. Fewer than 10 participants remain to be assigned to a treatment group. The ongoing COMBAT-ALS trial (NCT04057898) aims to evaluate…
A new coalition aims to use artificial intelligence (AI) tools to accelerate drug discovery for amyotrophic lateral sclerosis (ALS) and other neurodegenerative disorders. Answer ALS is teaming with partners including GATC Health — a drug discovery company focusing on machine learning and neural networks — Louisiana State…
AB Science has been cleared to start its confirmatory Phase 3 trial of masitinib for people with amyotrophic lateral sclerosis (ALS) in the U.S. and certain European countries. The company announced plans for the Phase 3 AB23005 trial earlier this year, noting then that the trial…
It’s hard to believe my husband, Todd, has been living with ALS for over 15 years. His limbs are completely paralyzed, and he uses a noninvasive ventilator nearly 24/7. He can still talk and eat, though not without difficulty. When people with ALS — or those who have a…
NKGen Biotech said it’s been cleared to launch an expanded access program (EAP) that will allow patients in the U.S. with neurodegenerative conditions, including amyotrophic lateral sclerosis (ALS), to access its experimental therapy troculeucel outside clinical trials. EAPs, or compassionate use programs, allow people with serious or life-threatening conditions…
The ALS Association has announced the winners of its first-ever Hoffman ALS Clinic Development and Capacity Awards, two new grant programs aimed at improving access to multidisciplinary care for people with amyotrophic lateral sclerosis (ALS) across the U.S. The awards are supported by a $58 million donation from the…
The ongoing program that is providing amyotrophic lateral sclerosis (ALS) patients with access to Medicinova’s investigational therapy MN‑166 (ibudilast) outside of a clinical trial is adding new sites. The expanded access program (EAP) will enroll about 200 patients at several locations in the U.S. who are not eligible…
AUT00201, an experimental oral small molecule being developed by Autifony Therapeutics, reduced the loss of motor neurons — the nerve cells that control movement — and eased motor symptoms in a mouse model of amyotrophic lateral sclerosis (ALS), a new study showed. It was also found to improve…
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