Researchers have discovered a pathway in cells that prevents the formation of TDP-43 protein clumps, which contribute to nerve cell damage in nearly all people with amyotrophic lateral sclerosis (ALS). The findings may aid ALS therapies that can activate the pathway and potentially limit TDP-43 clumping and prevent nerve…
A neurosurgeon and his team from the University of California (UC) Davis Health have won The Herbert Pardes Clinical Research Excellence Award — given by the nonprofit Clinical Research Forum — for their work on a brain-computer interface that translates brain signals into speech with greater than 95% accuracy.
Brainstorm Cell Therapeutics is moving forward in its efforts to get needed regulatory clearances to launch a Phase 3b clinical trial in the U.S. to test its stem cell therapy NurOwn (debamestrocel) in people with amyotrophic lateral sclerosis (ALS) with less advanced disease. The company has submitted to…
We didn’t realize how much my husband Todd’s comfort depended on his custom-fit power wheelchair until he had to go without it. A few weeks ago, Todd, who has ALS, decided to order a new wheelchair after finding out that his 11-year-old Permobil C300 isn’t fully compatible…
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…
