Simply Stated: What is Myasthenia Gravis (MG)?

Myasthenia gravis (MG) is a chronic neuromuscular disease characterized by muscle weakness that worsens after activity and improves after rest. MG is caused by an autoimmune reaction in which the body’s immune system attacks its own tissues, interrupting the connection between nerves and muscles (the neuromuscular junction). MG can occur at any age and affects . . .

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Simply Stated: What Is Neuromuscular Disease?

Neuromuscular disease refers to a diverse group of rare conditions that affect some part of the neuromuscular system, such as: Muscles Nerves in the peripheral nervous system (arms and legs) Nerves in the central nervous system (spinal cord) The neuromuscular junction, where nerves and muscles meet Neuromuscular diseases generally cause muscle weakness, fatigue, and other . . .

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Eledon Announces Positive Topline Results from a Phase 2a Study of Tegoprubart to Treat ALS

On May 31, Eledon announced positive topline results from its Phase 2a clinical trial of the investigational therapy tegoprubart (formerly AT-1501) for treatment of amyotrophic lateral sclerosis (ALS). The primary endpoints of the study, assessment of safety and tolerability, demonstrated an encouraging safety profile that supports further investigation. Secondary endpoints showed that the drug engaged . . .

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Simply Stated: What is Friedreich’s Ataxia (FRDA)?

Friedreich’s ataxia (FRDA) is an inherited neuromuscular disease that primarily impacts the nervous system and heart and affects about one in 50,000 people worldwide. FRDA typically appears in people before the age of 25 years and is characterized by a slow, progressive loss of limb coordination (ataxia) and effects on speech and swallowing. While there . . .

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Research Study Alert: Track-ALS Study of Home-Based Digital Monitoring to Assess ALS Progression

Researchers at the Barrow Neurological Institute (BNI) and Emory University are seeking adults living with amyotrophic lateral sclerosis (ALS) to participate in the Track-ALS research study to assess whether measurements collected at home, using digital applications, are dependable and can help with tracking disease progression. Study participants will be asked to perform various at-home tests at . . .

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Clinical Trial Alert: Phase 3 Study of investigational drug in Adults Living with ALS

Researchers at Cytokinetics, Inc. are seeking adults living with amyotrophic lateral sclerosis (ALS) to participate in the phase 3 COURAGE-ALS clinical trial to evaluate the safety and effectiveness of the investigational drug. In ALS, motor neurons (nerve cells that control muscle cells) are gradually lost, causing the skeletal muscles they control to become weak and nonfunctional. . . .

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Brain-Computer Interface Technology Could Improve Life for Millions

It’s been half a century since University of California, Los Angeles, computer science professor Jacques Vidal coined the term “brain-computer interface” (BCI) and produced the first peer-reviewed papers on the idea of interpreting brain signals with a computer. Today, BCI offers huge potential to improve the lives of millions of people around the world, including . . .

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Research Study Alert: Study of Wearable Gait Sensor in People with or without Neuromuscular Disease

Researchers at the University of California, Davis Neuromuscular Research Lab are seeking individuals with or without neuromuscular disease (ages 2 years or older) to participate in a research study to test the feasibility of using a wearable gait sensor to collect data as part of a new outcome measure for use in future clinical trials.  This . . .

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FDA Approves Alexion’s Ultomiris for Treatment of gMG

On April 28, the US Food and Drug Administration (FDA) granted approval to ravulizumab (Ultomiris) for the treatment of generalized myasthenia gravis (gMG) in adults who test positive for the anti-acetylcholine receptor (AChR) antibody. To date, Ultomiris is the third disease modifying drug approved to treat gMG. Ultomiris will be made available in the United . . .

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