Join the RASCALS Foundation for ALS Awareness Month at the big 5K Walk and Run at Tower Grove Park in St. Louis, Sunday May 4, 2014.
Posts belonging to Category ALS News
In a new study published in Nature Genetics, University of Pennsylvania researchers and colleagues have made inroads into the mechanism by which ALS acts. Working with a powerful fruit fly model of the disease, they found a way of reducing disease toxicity that slows the dysfunction of neurons and showing that a parallel mechanism can reduce toxicity in mammalian cells. Their discoveries offer the possibility of a new strategy for treating ALS.
While the size of this study is small, the ability of the specific biomarkers used to predict ALS prognosis suggests that the approach holds promise.
Traditionally, ALS patients like Mary would use a BiPAP machine to help her breathe. She’d wear a mask, tethered to a ventilator. It would force air into Mary’s lungs. Now, Mary is the first ALS patient at The Ohio State University to be implanted with a diaphragmatic pacing system to help her strengthen her muscles.
Research led by King’s College London has identified a new genetic variant, located on chromosome 17, associated with sporadic amyotrophic lateral sclerosis (ALS) – the most common form of motor neurone disease (MND).
For patients with amyotrophic lateral sclerosis (ALS), the simple act of breathing is often a tough thing to do. Now, these patients have new hope, as a device has been approved by the U.S. Food and Drug Administration (FDA) that may help patients to breathe more easily without a ventilator.
Respiratory failure is one of the major complications associated with amyotrophic lateral sclerosis (ALS). The quest for alternative approaches for treating respiratory failure has led to the study, and subsequent FDA approval, of diaphragm pacing for use in ALS patients.
“We are very excited to announce the selection of IMS-088 as ImStar’s lead ALS compound. As we continue with preclinical development next year, we hope to establish IMS-088 as a safe and effective new drug candidate for ALS,” said Jean-Pierre Julien, ImStar’s chief scientific officer.
According to a post hoc analysis, a greater percentage of patients receiving NP001 experienced a halt in disease progression which reached statistical significance when compared to the combination of concurrent and matched historical (placebo) controls. The high dose of NP001 (2mg/kg) halted progression in 27% of patients compared to 11% of patients on placebo. Further, NP001 was found to be safe and well-tolerated in the study.
Cytokinetics will elaborate on the clinical trial design and include enrollment and baseline demographics data from BENEFIT-ALS (Blinded Evaluation of Neuromuscular Effects and Functional Improvement with Tirasemtiv in ALS), which is evaluating tirasemtiv, a novel mechanism skeletal muscle activator, as a potential treatment for patients with amyotrophic lateral sclerosis (ALS).