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…
Denali Therapeutics‘ oral candidate DNL343 has failed to reduce levels of an established biomarker of nerve cell damage in adults with amyotrophic lateral sclerosis (ALS). That’s according to new data from the DNL343 arm of the HEALEY ALS platform trial (NCT04297683), where patients who received the experimental…
A bipartisan team of lawmakers in the U.S. House of Representatives and Senate is pushing for legislation to help ensure that spouses of veterans who die from amyotrophic lateral sclerosis (ALS) have access to benefits. The Justice for ALS Veterans Act was introduced in the House by Brian Fitzpatrick,…
Pathmaker Neurosystems has received three new patents — two in the U.S. and one in Japan — that cover the use of its noninvasive neuromodulation technology in people with amyotrophic lateral sclerosis (ALS). The patents, which specifically cover the systems and methods used for treating ALS and related diseases,…
The National Organization for Rare Disorders (NORD) is seeking participants for its survey-based study Living Rare, which aims to better understand the real-world lived experiences of people in the U.S. with rare diseases. Living Rare, the first large-scale study of its kind in the U.S., seeks to capture the…
Tiziana Life Sciences is seeking regulatory clearance in the U.S. to launch a Phase 2 clinical trial evaluating foralumab nasal spray, its lead therapeutic candidate, as a treatment for amyotrophic lateral sclerosis (ALS). The filing of the company’s investigational new drug application with the U.S. Food and Drug…
Woolsey Pharmaceuticals has extended its Series B preferred financing round, securing additional funds to continue advancing its ROCK inhibitor Bravyl (oral fasudil) for amyotrophic lateral sclerosis (ALS). The extension was completed with participation from existing investors. Series B financing is an early round of funding that usually takes…
Health Canada has conditionally approved Qalsody (tofersen) for the treatment of adults with amyotrophic lateral sclerosis (ALS) associated with mutations in the SOD1 gene. Qalsody, designed to lower levels of the toxic protein that drives the disease for these patients, is the first treatment to directly target the…
Note: This story has been updated March 6, 2025, to correct a quote from Mindy Henderson, MDA vice president of disability outreach and empowerment. The Muscular Dystrophy Association (MDA) is once again hosting its annual gathering, the MDA Clinical & Scientific Conference. The 2025 event will take place…
Measuring blood levels of neurofilament light chain, or NfL, a biomarker of nerve damage, may help in diagnosing amyotrophic lateral sclerosis (ALS), and in predicting the risk of death among people with the neurodegenerative condition, a new study shows. This biomarker showed substantially better accuracy for both ALS diagnosis…
