News

Qalsody slows ALS disease progression in real-world settings

Treatment with Qalsody (tofersen) in the real world slowed disease progression in people with amyotrophic lateral sclerosis (ALS) caused by SOD1 mutations, a small study reports. It also stabilized patients’ quality of life and lowered levels of nerve damage-related biomarkers, which is consistent with clinical trial data that…

Certain spinal fluid biomarkers may predict responses to NurOwn

Treatment with NurOwn (debamestrocel), which BrainStorm Cell Therapeutics is developing for amyotrophic lateral sclerosis (ALS), may bring about changes in biomarkers of inflammation and neurodegeneration that predict clinical outcomes. The findings come from the Phase 3 trial (NCT03280056) that tested NurOwn against a placebo in 189 adults with rapidly…

AAN 2024: Troriluzole may be taken with food, data suggest

Troriluzole, a new formulation of the approved amyotrophic lateral sclerosis (ALS) treatment riluzole, can be taken with or without food and appears to have better pharmacological properties than approved versions of the medication that allow once daily administration. That’s according to data from three studies in healthy volunteers presented…

Columbia gets $15M to design gene therapies for rare ALS forms

Columbia University researchers will use a $15 million federal grant to design individualized gene therapies for nine people with ultra-rare genetic forms of amyotrophic lateral sclerosis (ALS). The three-year grant comes from the National Institute of Neurological Disorders and Stroke’s (NINDS) Ultra-rare Gene-based Therapy (URGenT) Network, a program…

It’s year 70 for ‘Fill the Boot’ MDA fundraisers nationwide

The Muscular Dystrophy Association (MDA) is marking its seven-decade partnership with the International Association of Fire Fighters (IAFF) with this year’s launch of Fill the Boot, a nationwide yearlong fundraising campaign supporting research, care, and advocacy for people with neuromuscular disorders such as amyotrophic lateral sclerosis (ALS).