Focus of NORD study: Rare disease community’s lived experiences

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…

It’s become exceedingly difficult for my husband, Todd, to leave our home now that he’s in an advanced stage of ALS. His neck is weak because of disease progression, and he uses noninvasive ventilation nearly all the time. To make matters worse, we live in a northern climate with…

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…

As a former pastor and chaplain, Deb found the strength to transform the fear and frustration from her diagnosis into hope and help for others with ALS. Discover how Deb learned to celebrate every new day as a gift.

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…

Fat molecules called polyunsaturated fatty acids, or PUFAs, can help lessen nerve damage in amyotrophic lateral sclerosis (ALS), according to a study of fruit flies and cell models. Previous studies have shown that consumption of omega-3 fatty acid, a type of PUFA, is associated with a lower risk…

Lines from a Shania Twain song have been running through my head the past few days: “You’re still the one I run to/ The one that I belong to/ You’re still the one I want for life.” The lyrics remind me of how my husband, Todd, and I…