How new gene therapies are transforming genetic testing for ALS

A feature documentary airing on PBS delves into a man’s final years living with amyotrophic lateral sclerosis (ALS), offering an intimate portrait of resilience, caregiving, and enduring love while exploring how culture and identity shaped his experience with the disease. The film “…

I’ve been busy learning how to drive! No, not a four-wheeled automobile — it’s a six-wheeled power wheelchair. Although I continue to rely on a rollator to help me walk inside the house and a mobility scooter when I’m out and about, getting the power wheelchair was a strategic,…

A post-marketing analysis of safety reports found that many of the strongest signals involving Qalsody (tofersen) in people with amyotrophic lateral sclerosis (ALS) were related to the spinal procedures used to administer it. Other reporting signals involved neurological and inflammatory events. The study, based on 409 reports submitted…

A self-balancing, hands-free robotic exoskeleton designed for people with severe gait impairment was safe and well tolerated in a small pilot study of amyotrophic lateral sclerosis (ALS) patients who could still walk. While six weeks of walking practice with the Atalante exoskeleton did…

Targeting a specific part of the TDP-43 protein can prevent toxic protein clumping that drives amyotrophic lateral sclerosis (ALS) without disrupting the protein’s normal function, a new study shows. “Current [U.S.-approved] treatments for ALS provide only modest benefits. There is an urgent need for a real breakthrough,” Xinglong Wang,…

An expanded access program (EAP) at Massachusetts General Hospital (MGH) shows that such programs can work, a study found. The program enabled 11 people with SOD1-associated amyotrophic lateral sclerosis (ALS) who could not participate in clinical trials to receive Qalsody (tofersen) before its U.S. approval. In addition, it…

More than one in 10 people with amyotrophic lateral sclerosis (ALS) harbor at least one known disease-causing mutation, according to a new profile from the Strategic ALS Australia Systems Genomics Consortium (SALSA-SGC). The multi-site research consortium brings together major ALS clinics across Australia to collect clinical data and biological…