Mutations in the C9ORF7 gene, the most common genetic cause of amyotrophic lateral sclerosis (ALS), lead to defects in the stem cells needed for brain development in the early stages of life, a new study shows. In animal models of the disease, this resulted in smaller than normal volumes…
ALS mutation causes brain stem cell defects at early development
We had a young visitor this past weekend who met my husband’s disability with frank curiosity and innocent sweetness. The guest, Todd’s 6-year-old great-nephew, watched with interest as I set up his Uncle Todd at the computer. “What is that for?” he asked as I put Todd’s sip-and-puff…
Treatment with CNM-Au8 was found to significantly delay disease progression and prolong survival, by more than 1.5 years, among adults with early-stage amyotrophic lateral sclerosis (ALS) in the RESCUE-ALS trial and its open-label extension phase. That’s according to two different statistical analyses, which overall showed that patients who…
A new two-year, $400,000 grant will support studies — by EverythingALS and the Sean M. Healey and AMG Center for ALS at Massachusetts General Hospital (MGH) — aimed at identifying early digital diagnostic markers in amyotrophic lateral sclerosis (ALS). The award, from ALS Finding a Cure…
A number of years ago, during my first year of learning to live with ALS, I promised myself that I’d show up every single day. At that time, my notion of showing up meant my goal was simply to get up, get dressed, and go sit in the…
Scientists have devised a technique for transplanting healthy motor neurons — the nerve cells that are lost in amyotrophic lateral sclerosis (ALS) — in a mouse model with “highly aggressive” disease. Transplanted motor neurons, given stimulation, were able to form healthy connections with muscle cells to control the animals’ muscle…
Akava Therapeutics will soon launch a Phase 1 clinical trial to test AKV9, its investigational therapy for amyotrophic lateral sclerosis (ALS), in healthy people. The trial follows the clearance of Akava’s investigational new drug (IND) application by the U.S. Food and Drug Administration (FDA). It will be AKV9’s…
My late husband, Jeff, made a dream come true for me. When I was a kid, my dad would take our family on our small powerboat to explore the waters near my hometown of Annapolis, Maryland. If we had enough time and the weather cooperated, we’d take a…
While eating a high-calorie diet is safe and well tolerated, it does not appear to slow disease progression or reduce the risk of death in people with amyotrophic lateral sclerosis (ALS), a review of published clinical trials found. Combining data from multiple trials helps to create a single, more…
One evening last week after I’d finished cooking dinner, I grew concerned. My almost 14-year-old son had gone out to ride his dirt bike on an off-road vehicle (ORV) trail that ran next to our property. He’d planned to go only to a nature area a couple miles away and…
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