Treatment with the cell therapy NurOwn may slow the progression of amyotrophic lateral sclerosis (ALS) for those with a specific variation in the UNC13A gene that is linked with a high risk of the disease. Merit Cudkowicz, MD, chief of neurology at the Massachusetts General Hospital, shared the…
#MDA2022 – NurOwn May Benefit Patients With Specific Gene Variant
Treatment with Clene Nanomedicine’s experimental medicine CNM-Au8 may decrease the risk of disease progression or death in people with early amyotrophic lateral sclerosis (ALS), according to new analyses from the RESCUE-ALS clinical trial. Data also suggest that the therapy helped to prevent the loss of motor neurons in…
I felt twinges of grief as I shopped for a card for a young couple’s wedding. First card: “Wishes on this special day for happiness, joy, and love, along with a future that will bring everything you are dreaming of.” Sigh. I wish my husband and I had a future.
Columns
The Same as It Never Was
“And you may find yourself living in a shotgun shack … And you may ask yourself, ‘Well, how did I get here?’ … Letting the days go by, same as it ever was.” Although I’m not proud of it, my life, pre-ALS, was largely a fortunate stream of autopilot idleness.
An ambitious Irish research project, called Precision ALS, will combine clinical research, data science, and artificial intelligence (AI) to identify the multiple — and potentially targetable — factors involved in the development and progression of amyotrophic lateral sclerosis (ALS). The goal is to use this information to determine which treatments…
If you happened to read my column last week, you’re probably thinking I’m writing this while sitting on a sunny beach, gazing at the ocean, and sipping a tropical drink with a little paper umbrella in it. That’s because I wrote about my busy month filled with ALS-related events,…
Exposure to certain toxic compounds — including beta-N-methylamino-L-alanine (BMAA), formaldehyde, and heavy metals like manganese, mercury, and zinc — increases the likelihood of developing amyotrophic lateral sclerosis (ALS), according to a review paper. Its authors argue that with BMAA — a toxic compound made by algae — enough evidence…
Patient registries are a hot topic of rare disease research and many organizations are taking advantage of this resource by signing up their patient communities and connecting with researchers. Eric Sid, MD, program officer for the Office of Rare Diseases Research (ORDR), said it is difficult to estimate how…
University of Sydney neurologist Matthew Kiernan, PhD, has been granted the 2022 Sheila Essey Award for his groundbreaking research, aimed at providing earlier diagnoses and better treatments for people with amyotrophic lateral sclerosis (ALS). Kiernan, a world-leading expert in the field of ALS research, is the first Australian to…
A Phase 1 clinical trial investigating ProJenX‘s oral therapy prosetin in healthy volunteers and people with amyotrophic lateral sclerosis (ALS) has started dosing participants. The first-in-human PRO-101 trial is divided into three parts. In parts 1a and 1b, researchers will investigate the safety, tolerability, and pharmacokinetics of single and…
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