Amyotrophic Lateral Sclerosis News and Research

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Advances in chemistry and delivery are taking oligonucleotide medicines beyond rare diseases

A review in Cell Reports Medicine examines how advances in oligonucleotide chemistry and delivery have established ASOs and RNA interference therapies as a class of genetic medicines, with 21 oligonucleotides approved at the time of writing. It also identifies extrahepatic delivery, safety, target validation, individualized treatment, and data quality as priorities as the field expands into common diseases and explores AI-assisted development.

24 Sep 2026

Experimental drug reverses multiple sclerosis damage in mouse model

A drug developed by a UVA Health scientist has not only stopped but reversed paralysis and vision loss in a mouse model of multiple sclerosis.

23 Sep 2026

Genetic system converts scar-forming cells into neurons to restore movement after spinal cord injury

What if cells that form scars after a spinal cord injury could become part of the repair process? Researchers have developed a genetic system that converts certain scar-forming cells into neurons, improving movement in mice and rats with spinal cord injuries.

23 Sep 2026

New discovery overturns a prevailing model of brain development

For centuries, scientists have thought of the brain as a single, unified organ. But new research led by Stanford Medicine reveals that what we call the brain is two distinct organs that evolved independently over hundreds of millions of years.

18 Sep 2026

One brain implant could give people with paralysis a more expressive way to communicate

Researchers showed that a single high-density ECoG implant could decode attempted speech and upper-body gestures in parallel in people with severe paralysis, allowing two participants to control a personalized full-body avatar. Training the decoders on both isolated and concurrent behaviors improved performance across contexts and reduced false activations, offering proof-of-concept evidence for more expressive multimodal BCIs.

16 Sep 2026

Study uncovers mechanism for reading extra-long neuronal genes

Neurons in humans and other mammals express many exceptionally long genes, ranging from more than 100 kb to over 2 Mb, that are important for forming synapses and neural circuits.

15 Sep 2026

AI platform uncovers new molecular targets for Alzheimer's disease

Groundbreaking study published in Alzheimer's & Dementia reveals the crucial links between the NAD⁺-mitophagy axis and neurodegeneration.

1 Sep 2026

Compound PF-04457845 slows motor decline in ALS mouse models

Amyotrophic lateral sclerosis (ALS) is a severe neurological disease that destroys nerve cells responsible for muscle movement.

28 Aug 2026

Shared protein pathology redefines treatment for multiple neurodegenerative diseases

On the surface, amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD) and limbic-predominant age-related TDP-43 encephalopathy (LATE) might seem like unrelated diseases.

24 Aug 2026

Disrupted brain-immune signaling may help drive neurodegeneration

A Cell perspective argues that neurodegeneration can emerge from disrupted communication between neural and immune networks, with peripheral immunity, microglia, and gene-regulatory programs all shaping disease processes. The authors propose that restoring immune homeostasis and recalibrating neuroimmune circuits could create new therapeutic and diagnostic opportunities, while emphasizing that many mechanisms remain unresolved in humans.

24 Aug 2026

A single blood test could offer a glimpse of disease risk years in advance

Machine learning-derived cellular aging clocks mapped more than 7,000 plasma proteins to over 40 cell types, revealing marked differences in how individual cell populations age. These blood-based signatures were associated with future risks of Alzheimer’s disease, ALS, lung cancer, type 2 diabetes, and mortality across follow-up extending to 15 years.

19 Aug 2026

New review urges proteostatic fitness standards for stem cell therapies

Stem cell therapies have expanded rapidly, with more than 115 clinical trials involving over 1,200 patients approved by late 2024. The U.S. Food and Drug Administration (FDA) recently approved Ryoncil (remestemcel‑L) for children with steroid‑refractory acute graft‑versus‑host disease, signaling a major shift toward routine clinical use.

11 Aug 2026

Occupational pesticide exposure may significantly increase ALS risk

Workplace exposure to pesticides (herbicides and insecticides) is linked to a 60-70% heightened risk of the most common form of motor neuron disease-amyotrophic lateral sclerosis, or ALS for short, finds a synthesis of the available evidence, published online in Occupational & Environmental Medicine.

4 Aug 2026

Researchers recreate imagined melodies from brain activity

In a study led by Jii Kwon and Chun Kee Chung, from Seoul National University, researchers explored whether they could recreate melodies from the brain activity of people imagining how songs continue after listening to a few bars.

3 Aug 2026

ALS risk varies significantly across U.S. military service branches

Compared to Army veterans, men who are veterans of the U.S. Air Force, Navy and Coast Guard have higher rates of amyotrophic lateral sclerosis (ALS), and men who are Marine veterans have lower rates, according to a new study published July 29, 2026, in Neurology®, an official journal of the American Academy of Neurology.

29 Jul 2026

Photoreceptors possess internal recycling system to preserve vision

For decades, scientists believed that photoreceptors in the retina relied primarily on neighboring support cells, known as the retinal pigment epithelium (RPE), to remove waste. While the RPE is still responsible for clearing away the worn-out tips of photoreceptors each day, new findings from the University of Oklahoma reveal that photoreceptors also perform much of their own internal housekeeping.

29 Jul 2026

New blood protein biomarkers estimate timing of ALS symptoms

Months to several years before amyotrophic lateral sclerosis (ALS) symptoms arise, levels of certain blood proteins may dramatically shift.

27 Jul 2026

Walking protein structure explains how cells activate molecular motors

A nerve cell resembles a vast tree with branches that communicate with thousands of other cells. To function, it depends on a motor protein that walks on two legs, hauling urgent cargo from the center of the cell to the faraway tips of every branch.

15 Jul 2026

Advancing Motor Neuron Disease Diagnosis with Non-Invasive Ultrasound and AI

Research led by Professor Hodson-Tole combines ultrasound and AI to improve motor neuron disease diagnosis, reducing reliance on invasive procedures.

6 Jul 2026

Experimental drug shields nerve cells from damage caused by ALS

Amyotrophic Lateral Sclerosis, or ALS, received widespread awareness in the summer of 2014 thanks to an Ice Bucket Challenge. Millions of people participated in the challenge, drenching themselves in ice cold water to bring visibility to this devastating neurodegenerative disease and generate charitable donations for research.

3 Jul 2026