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Experimental drug shows early promise against inherited form of ALS, trial...

A clinical trial has found evidence that the experimental drug tofersen lowers levels of a disease-causing protein in people with an inherited form of amyotrophic lateral sclerosis, or ALS, caused by...

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Scientists identify promising new ALS drug candidates

Scientists have taken a significant step forward in the search to find effective new drug candidates for the treatment of Amyotrophic lateral sclerosis (ALS), also known as motor neuron disease.

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Investigational drug stops toxic proteins tied to neurodegenerative diseases

An investigational drug that targets an instigator of the TDP-43 protein, a well-known hallmark of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), may reduce the protein's...

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Faulty transportation of messenger RNA is the culprit in ALS

Researchers have discovered a function for the protein missing in many types of ALS and FTLD, two neurodegenerative diseases. In neurons, the protein TDP-43 bound to messenger RNA that codes for pieces...

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Investigational ALS drug prolongs patient survival in clinical trial

An experimental medication that was recently shown to slow the progression of amyotrophic lateral sclerosis has now demonstrated the potential to also prolong patient survival. The findings come from a...

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New biomarker candidate for amyotrophic lateral sclerosis

A research team has developed a diagnostic tool for the rare neurological disease amyotrophic lateral sclerosis (ALS). The study used the patented immuno-infrared sensor to analyze folding changes of...

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ALS study reveals a unique population

The secrets long hidden in the DNA of patients with amyotrophic lateral sclerosis (ALS) living in Malta have been unlocked, according to a new study.

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ALS neuron damage reversed with new compound

Scientists have identified the first compound that eliminates the ongoing degeneration of upper motor neurons that become diseased and are a key contributor to ALS (amyotrophic lateral sclerosis), a...

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Study provides insights into architecture of abnormal protein deposits in...

Scientists have determined the structure of protein 'fibrils' linked to Lou Gehrig's disease and other neurodegenerative disorders -- findings that provide clues to how toxic proteins clump and spread...

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Motor neurons derived from patients point to new possible drug target for ALS

Researchers now report a high-throughput target and drug discovery platform using motor neurons made from ALS patients. Using the platform, they confirmed two known targets and identified an existing...

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A distinctive inflammatory signature found in a genetic form of ALS

Researchers find an increase in inflammatory molecules in serum and cerebrospinal fluid of C90RF72 patients, informing future anti-inflammatory therapies.

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Discovery of the role of a key gene in the development of ALS

Amyotrophic lateral sclerosis, or ALS, attacks nerve cells known as motor neurons in the brain and spinal cord, gradually leading to paralysis. The loss of function of an important gene, C9orf72, may...

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Research uncovers new insights on ALS and points to a potentially promising...

Investigators have found that an abnormal form of the tau protein is present in brain tissue from deceased patients who had amyotrophic lateral sclerosis (ALS). Cells that were grown in contact with...

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Researchers discover new insights about tau proteins in people living with ALS

Investigators found elevated levels of tau and a phosphorylated form of the tau protein in brain tissue from deceased people who had amyotrophic lateral sclerosis (ALS) and carried a mutation in the...

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Repurposed ALS drug shows promise in mouse model of rare childhood genetic...

Riluzole, a drug approved to treat amyotrophic lateral sclerosis (ALS), a disease affecting nerve cells controlling movement, could slow the gradual loss of a particular brain cell that occurs in...

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ALS therapy should target brain, not just spine

The brain is indeed a target for treating ALS (amyotrophic lateral sclerosis), scientists have discovered. This flips a long-standing belief the disease starts in the spinal motor neurons and any...

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Detailed images of molecule associated with ALS could open door to therapies

Scientists have determined the structure of the molecule associated with amyotrophic lateral sclerosis (ALS) and multiple other neurodegenerative diseases. The researchers say this discovery could...

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Astrocyte studies reveal harmful changes in amyotrophic lateral sclerosis

Scientists have revealed harmful changes in supporting cells, called astrocytes, in amyotrophic lateral sclerosis (ALS).

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New meta-analysis explores potential environmental causes of ALS disease

In a new meta-analysis of available ALS literature, researchers explore environmental influences potentially linked ALS disease, using rigorous quantitative methods. The study also examines the...

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Discovery of a new ALS and dementia disease mechanism raises treatment hopes

A pioneering new study has revealed, for the first time, why a common genetic variant worsens disease outcomes for people with the devastating adult-onset neurodegenerative diseases amyotrophic lateral...

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Scientists discover a new molecular pathway shared by two neurodegenerative...

Researchers from two independent research teams have discovered how the mislocalization of a protein, known as TDP-43, alters the genetic instructions for UNC13A, providing a possible therapeutic...

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Potential window for treating ALS identified

Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disorder that affects as many as 30,000 people in the United States, with 5,000 new cases diagnosed each year. It weakens muscles over time,...

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New ALS 'drug' is more effective than existing ones

New preclinical research on the experimental drug, NU-9, to treat ALS shows it is more effective than existing FDA-approved drugs for the disease. More importantly, NU-9 has an enhanced effect when...

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Muscle biopsy test for biomarker could lead to earlier diagnosis of ALS

Amyotrophic lateral sclerosis (ALS) is a progressive disease of the nervous system. It affects nerve cells in the brain and spinal cord called motor neurons. Motor neurons control muscle movement and...

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ALS may be linked to both the immune and central nervous systems

The immune system may play a fundamental role along with the central nervous system in amyotrophic lateral sclerosis (ALS), also known as 'Lou Gehrig's disease,' researchers report.

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First mouse model with mitochondrial tRNALeu mutation developed

Researchers have developed the first mouse model for a mitochondrial tRNALeu mutation, showing that the associated metabolic disorder results from faulty RNA processing.

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Promising MND drug helps slow disease progression and benefits patients...

Scientists believe a new genetically-targeted therapy to treat motor neurone disease (MND) could be a turning point for patient care, after the results of a Phase 3 clinical trial showed significant...

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Investigational drug for genetic form of ALS improves disease's molecular signs

An international phase 3 clinical trial for a rare, inherited form of ALS showed that an investigational drug, known as tofersen, reduced molecular signs of disease, but at six months did not improve...

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New analysis of cellular 'vehicles' drives a deeper understanding of ALS,...

Scientists have taken a key step toward better understanding neurodegenerative diseases by using a suite of biophysical techniques to learn more about a motor protein whose malfunction is associated...

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Reading out RNA structures in real time

A new microscopic technique allows for the real-time study of RNA G-quadruplexes in living cells, with implications for the fight against amyotrophic lateral sclerosis.

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RNA modification 'pivotal' for protein linked to neurodegeneration in ALS

Scientists know that TDP-43, which helps regulate processing of RNA, may be responsible for the death of nerve cells in ALS and frontotemporal dementia. And a study suggests that a common modification...

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Researchers discover therapeutic target to aid in glaucoma treatment

Researchers have identified a new therapeutic target that could lead to more effective treatment of glaucoma.

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DNA treatment could delay paralysis that strikes nearly all patients with ALS

Researchers use a DNA designer drug to restore key protein levels in motor neurons, delaying paralysis in a mouse model of ALS.

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Parkinson's disease drug ropinirole safely slowed the progression of ALS for...

Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, is a fatal motor neuron disease that causes people to gradually lose control of their muscles. There is no cure, and current...

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Research reveals the scale of disorder underpinning Motor Neurone Disease

Researchers have shown that hundreds of proteins and mRNA molecules are found in the wrong place in nerve cells affected by Motor Neuron Disease (MND), also known as Amyotrophic Lateral Sclerosis (ALS).

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New insights into the protein-mediated motor neuron loss in amyotrophic...

Amyotrophic lateral sclerosis (ALS) is a widespread neurodegenerative disease caused by the loss of neurons that control movement. Although abnormal accumulation of proteins called TDP-43 accompanies...

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Guidelines follow advances in ALS gene discovery, ongoing gene therapy trials

Researchers have developed an evidence-based consensus guidelines for genetic testing and counseling for patients with amyotrophic lateral sclerosis (ALS), a neurodegenerative disease that affects the...

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A promising drug candidate for ALS -- prolongs lifespan and eases symptoms in...

A research group has found a promising drug candidate for the treatment of amyotrophic lateral sclerosis (ALS). Cerebral dopamine neurotrophic factor CDNF prolongs the lifespan of and alleviates...

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New therapeutic strategy to reduce neuronal death in amyotrophic lateral...

Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease that affects neurons in the brain and spinal cord causing loss of muscle control. Scientists have now suggested a potential...

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This is how protein aggregates can trigger neurodegenerative diseases

Neurodegenerative diseases are characterized by the deposition of clumped proteins in the brain and progressive neuronal cell death. Although the causal link between protein aggregates and...

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A molecular anchor

Scientists identify ALS-linked protein VAP as a key stabilizer of mitochondria near dendritic spines to support memory formation. This finding opens new directions for research into cognitive and motor...

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ALS: Blocking inflammation to reduce symptoms

In people with amyotrophic lateral sclerosis (ALS), changes in neurons appear to activate immune cells. Lowering the inflammation could reduce the symptoms of the disease, according to a new study.

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Pharmacological inhibitor protects nerve cells in ALS disease

A new pharmacological inhibitor can intervene in a central cell death mechanism that is responsible for the death of motor neurons and hence important for the progression of the motor neuron disease...

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Cracking the code of neurodegeneration: New model identifies potential...

Scientists have developed an innovative neural cell culture model, shedding light on the intricate mechanisms underlying neurodegeneration. Their research pinpointed a misbehaving protein as a...

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Recreational activities such as golfing, gardening may be associated with...

Participation in recreational activities -- including golfing, gardening or yard work, woodworking and hunting -- may be associated with an increase in a person's risk for developing ALS. Researchers...

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Integrity of blood-brain barrier depends on protein that is altered in some...

Defects in the blood vessel network of the central nervous system have been linked to early symptoms of neurodegenerative diseases such as Alzheimer's disease and amyotrophic lateral sclerosis (ALS)....

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New immune cell therapy benefits laboratory models of ALS and has some...

The study provides the basis for a phase I clinical trial in additional participants.

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Imaging technique uncovers protein abnormality in motor neurone disease

Researchers have used a new technique to identify pathological abnormalities associated with motor neurone disease.

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New brain-computer interface allows man with ALS to 'speak' again

A new brain-computer interface translates brain signals into speech with up to 97 percent accuracy. Researchers implanted sensors in the brain of a man with severely impaired speech due to amyotrophic...

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Two proteins identified as potential targets to improve ALS symptoms

Scientists have found that the protein histone H1.2 and the enzyme PARP1 could be potential therapeutic targets to decrease neurodegeneration in amyotrophic lateral sclerosis (ALS).

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