Last week for spring break, I traveled with my son from snowy Michigan to sunny Florida. Siesta Key was just as beautiful as I remembered from 15 years ago when my husband, Todd, and I vacationed there with our two small children, along with Todd’s parents and my brother…

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.

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…

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…

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…

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…

The Muscular Dystrophy Association (MDA) has rolled out its St. Patrick’s Day fundraising campaign, MDA Shamrocks — the largest such campaign in the U.S. marking this day — to raise money for research, patient care, and advocacy for amyotrophic lateral sclerosis (ALS) and other rare neuromuscular…

Four experimental treatments for amyotrophic lateral sclerosis (ALS) have been tested in the HEALEY ALS platform trial and two, CNM-Au8 and pridopidine, have shown promising results and are moving toward Phase 3 testing. The others, zilucoplan and verdiperstat, failed to show any benefit and are no…

It’s been a rough couple weeks since my husband, Todd, came down with a respiratory illness. The first weekend was the worst, with nausea, fever, chills, body aches, and chest congestion. By day four, he was able to eat a bit, and he spent longer stretches of the day…

A photo I came across this week from when my late husband, Jeff, was living with ALS flooded me with emotions. It was taken in November 2019, the month we moved into an accessible home. His ALS was advancing rapidly at the time, which prompted our move. He could…