Researchers have developed a type of gene therapy that can rescue the function of the TDP-43 protein in diseased nerve cells in amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases while leaving healthy cells untouched. The approach uses problematic sequences called cryptic exons that are only present in cells…
Highly targeted approach supports ALS gene therapy development
Neuvivo has filed an application with the U.S. Food and Drug Administration (FDA) requesting the approval of its investigational therapy NP001 (sodium chlorite) to treat amyotrophic lateral sclerosis (ALS). The new drug application comes on the heels of combined data from Phase 2a (NCT01281631) and Phase…
A growth factor called FGF4 can reduce the activity of astrocytes — star-shaped cells in the brain and spinal cord that become overly reactive in amyotrophic lateral sclerosis (ALS) — but it doesn’t stop motor neurons from dying, a study in cells and mice found. The researchers also observed…
Life’s curveballs happen to us all. Many of my column’s topics have been about the unexpected events I face while I live with ALS, as well as what I do to conquer them. But I believe the secret to my ability to handle life’s ups and downs is…
Differences in tear metabolites — the products of metabolism that are found in human tears — in people with amyotrophic lateral sclerosis (ALS) were able to distinguish bulbar-onset disease, in which symptoms initially affect the face and throat, from spinal-onset ALS, where the first symptoms usually are weakness in…
Researchers detected changes in the production of several immune signaling proteins, called cytokines, in the cerebrospinal fluid (CSF) — the liquid that surrounds the brain and spinal cord — of people with amyotrophic lateral sclerosis (ALS) at diagnosis. These changes also correlated with certain clinical characteristics, and how quickly the…
This morning, while my husband, Todd, was sleeping in the next room, I read a few chapters of Brian Jeansonne’s new memoir, “Onward Forward — My Journey with ALS,” in which he examines with vulnerability and insight his life with the disease. In Chapter 11, Jeansonne, a spiritual…
Modality.AI is teaming up with Target ALS on a clinical study of artificial intelligence (AI) as way to assess changes in speech and motor function in people with amyotrophic lateral sclerosis (ALS). The trial will harness a virtual, conversational AI system, called Tina, intended to detect even…
Neurosense Therapeutics announced that it has received a U.S. patent covering the formulation of PrimeC, its lead investigational therapy for people with amyotrophic lateral sclerosis (ALS) that combines two approved medications in a fixed dose. The patent, “Compositions comprising Ciprofloxacin and Celecoxib,” was issued by…
“What was I thinking?” The words boomeranged around my mind as I stood frozen in place under the hot Arizona sun. I was experiencing an ALS patient’s worst nightmare: being stuck with an expanse of sidewalk ahead of me, the same distance behind me, and absolutely zero energy in…
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