Twice-daily treatment with PTC Therapeutics’ oral candidate utreloxastat failed to significantly slow disease progression in adults with amyotrophic lateral sclerosis (ALS) in a global, placebo-controlled Phase 2 clinical trial. The results of CardinALS (NCT05349721) showed that, while the therapy was generally safe, it failed to meet its…
The University Health Network (UHN) in Toronto was selected as the first site in Canada to be part of a clinical trial testing Neuralink‘s brain-computer interface (BCI) in people unable to move their limbs due to amyotrophic lateral sclerosis (ALS) or spinal cord injury. The start of patient…
A Phase 1 clinical trial of QRL-201, Quralis‘ therapy candidate for amyotrophic lateral sclerosis (ALS), has successfully completed its dose-escalation phase, with the two doses tested reaching therapeutic levels in the spinal fluid of patients. The trial, dubbed ANQUR (NCT05633459), is now moving toward a dose range-finding…
People with mutations in the C9ORF72 gene who develop amyotrophic lateral sclerosis (ALS) show signs of damage to certain brain regions years before the appearance of disease symptoms, a study reports. The findings suggest that looking for changes in brain structures could help predict which people with C9ORF72 mutations…
As I drove through town, I cringed reading a church’s changeable-letter sign: “Be thankful for everything that happens to you. It’s all part of the experience.” There’s scientific evidence promoting the benefits of gratitude, so I try to practice it, but I get tired of counting my blessings…
Dear Santa, here’s my special wish list for the holiday season. I’m not asking for toys or special treats, just your help in making my holiday social events blend better with my life with ALS. Specifically, I’m hoping you can help me when I attend social gatherings, dine in…
Widetrial, an integrated service and technology platform, is coming on board to support an expanded access program (EAP) that will provide the investigational therapy MN-166 (ibudilast) to amyotrophic lateral sclerosis (ALS) patients who aren’t eligible for clinical trials. The program is funded under a $22 million federal…
Eli Lilly will develop therapies against two drug targets for amyotrophic lateral sclerosis (ALS) that were identified and validated by Verge Genomics as part of a three-year collaboration between the companies. The targets, which weren’t disclosed, were discovered with Converge, Verge’s platform that draws on human…
RAG-21, a therapy Ractigen Therapeutics is developing for amyotrophic lateral sclerosis (ALS) linked to mutations in the FUS gene, has been awarded orphan drug status by the U.S. Food and Drug Administration (FDA). Orphan drug status in the U.S. is granted to products aiming to prevent, diagnose, or treat…
I remember the first time my late husband, Jeff, fell as a result of ALS. Falls can be common with the disease, but I was still shocked each time one happened. When Jeff fell, it laid bare how ill qualified I felt to care for him, even while taking…
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