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…
MN-166 (ibudilast) is an experimental oral therapy that’s designed to reduce inflammation and protect nerve cells from damage in people with ALS. Developed by MediciNova, it’s expected to slow disease progression.
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…
The ALS Association has awarded a “groundbreaking” grant to Tiziana Life Sciences to support a small, early-stage clinical trial testing foralumab nasal spray for amyotrophic lateral sclerosis (ALS). The upcoming trial intends to investigate the safety and effectiveness of two foralumab doses in 20 ALS patients. It…
Answer ALS and Cedars-Sinai have made openly available their amyotrophic lateral sclerosis (ALS) patient-based stem cell and biodata repository to researchers worldwide. The largest of its kind, the repository contains biological and clinical data from nearly 1,000 ALS patients, as well as specialized nerve cells called induced pluripotent…
