A single dose of Insmed’s investigational gene therapy INS1202 preserved motor function and prolonged survival in a mouse model of amyotrophic lateral sclerosis (ALS) associated with SOD1 gene mutations. The therapy was also found to protect against nerve cell loss and had other beneficial effects on disease-related biomarkers.
MDA 2025: INS1202 gene therapy shows promise in SOD1-ALS mice
After decades of classic cross-country skiing, I realized that the groomed track is too wide for me, and it got me thinking how difficult it is to find single solutions to help everybody with ALS. We had springlike weather this past week in Michigan’s Upper Peninsula, and I got…
An experimental oral small molecule being developed by Quralis for amyotrophic lateral sclerosis (ALS) reduced motor neuron excitability as intended in healthy volunteers. That’s according to top-line data from a Phase 1 clinical study (NCT06681441) that tested two doses of QRL-101 against a placebo in healthy adults.
The phrase “stay in your lane” is often posted on signs to help motorists drive through a construction zone. It’s also a basic rule when swimming side by side in a race with other competitors. In my life with ALS, I use one of the other meanings of the…
Cognixion is recruiting adults with amyotrophic lateral sclerosis (ALS) for a clinical trial testing ONE Axon-R, a wearable headset that combines augmented reality and brain-computer interface technology with an artificial intelligence (AI) application to facilitate communication. Launched early this year at a single site in the U.S., the…
The ALS Society of Canada (ALS Canada) has named 39 Canadians to be presented the King Charles III Coronation Medal for their efforts into research and advocacy for amyotrophic lateral sclerosis (ALS). The Coronation Medal program, launched in 2023 to mark the coronation of His Majesty King…
Using a smartphone app to self-assess ALS Functional Rating Scale-Revised (ALSFRS-R) scores, a standardized measure of amyotrophic lateral sclerosis (ALS) severity, may be as reliable as clinic-based assessments, a study reports. Its findings suggest this app could be a useful digital tool for remote monitoring of ALS, allowing patients…
When I was growing up, many of my neighbors participated in the U.S. Naval Academy Sponsor Program, as we lived just a few miles from the school’s campus in Annapolis, Maryland. Under the program, local families “adopted” midshipmen, offering a home away from home to the students, who could come…
Treatment with CNM-Au8 in the HEALEY ALS platform trial prolonged survival for people with amyotrophic lateral sclerosis (ALS) relative to those who received the investigational treatment zilucoplan or a placebo in a separate arm of the study, according to new analyses. The benefits were most pronounced in the…
“I can’t believe I could’ve had longer footrests this whole time,” my husband, Todd, said this morning as he peered down on his wheelchair while I transferred him using our overhead lift. “I’ve been so nervous about my toes, especially now that I’m having such a hard time driving.” Todd…
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