The ALS Association joined 37 patient groups opposing a decision from a federal court in Texas that declared the Affordable Care Act (ACA) unconstitutional. The Texas v. United States decision is expected to be appealed to the U.S. Court of Appeals for the Fifth Circuit.
Dr. Brian Wainger from the Massachusetts General Hospital (MGH) presented initial top-level results from a recently completed phase II clinical trial of ezogabine (retigabine) on motor neuron excitability (NCT02450552). The study, supported by The ALS Association, met its main goal of quantifying a reduction in motor neuron excitability in people with ALS following treatment. Results were presented during the 29th International Symposium on ALS/MND in Glasgow, Scotland, last week.
I was diagnosed with ALS in January 2014, at the age of 53. I was an oncology nurse for 32 years and had just completed my master’s degree in nursing and passed the boards to become a nurse practitioner when I was diagnosed. Being a nurse practitioner had always been a dream of mine, but I was unable to use my master’s degree at all because of ALS.
People living with ALS come first in everything we do. We’re dedicated to providing people fighting ALS and their families and friends with the critical information, support, and resources they need to live full lives and better meet daily challenges.
In a promising new study by Drs. Robert Brown and Christian Mueller at the University of Massachusetts Medical School report that a type of viral gene therapy using synthetic microRNAs (miRNAs) targeting the ALS SOD1 gene is safe and effective in nonhuman primate macaques (monkeys). The ALS Association provided $1.7 million in funding for this study, which demonstrated an efficient reduction of the SOD1 protein without side effects. This paves the way forward for further development of this potential therapy.
Mitsubishi Tanabe Pharma America (MTPA) will present initial data on efforts to create an oral version of edaravone, a key drug in the treatment of ALS that is currently only available intravenously, during the International Symposium on ALS/MND in Glasgow, Scotland. The symposium will be held Dec. 7-9. MTPA is expected to present results that demonstrate that oral edaravone is processed in the body in the same manner as the infused formulation.
There’s no sugarcoating it. ALS is terrible. Unlike many fathers this Halloween, I couldn’t carve a pumpkin with my kids. I also couldn’t help them put on their costumes or take them trick-or-treating.
Why motor neurons die in ALS largely remains a mystery. In a study funded by The ALS Association, Dr. Anne Hart, professor of neuroscience at the Carney Institute for Brain Science at Brown University, and her colleagues gained insight into why some types of motor neurons die, while other don’t. A paper about the study was recently published in PLOS Genetics.
More than 1,200 participants, volunteers, and members of the community came together for a family-friendly day of celebration, exercise, education, awareness, music, food, and family fun at The ALS Association Golden West Chapter’s 14th annual Napa Valley Ride to Defeat ALS and Walk on Saturday, September 22.
Our national network of 63 Certified Treatment Centers of Excellence (CTCEs) provides robust, expert multidisciplinary ALS care and services in a supportive atmosphere, emphasizing hope and quality of life. Studies show attending a multidisciplinary clinic can extend survival, increase quality of life, and improve access to potential therapies.
Meet Dr. James Shorter, professor of Biochemistry and Biophysics at the Perelman School of Medicine at the University of Pennsylvania in Philadelphia. He and his team work tirelessly in the lab to better understand the causes of ALS, so those causes can be translated into potential therapeutic targets. The ALS Association has proudly funded him since 2014.
Dr. Emily Plowman, one of our Clinical Management Grant awardees, recently published a promising case report on a person with early-stage ALS who has been participating in a mild-intensity respiratory strength training program. The individual has been doing the strength training program with Dr. Plowman and her team at the University of Florida in Gainesville, Florida, over the past 24 months.
Yesterday, MediciNova, Inc. announced that the U.S. Food and Drug Administration (FDA) relayed positive feedback regarding its phase III clinical trial plan to test MN-166 (ibudilast) in a broad population of people with ALS. No safety issues were raised by the FDA and safety will be revisited when results are available in the phase III trial.
An amazing group of people came together for Climb to Defeat ALS earlier this month. The first Team Challenge ALS team summited Mt. Elbert in Colorado on September 7 and has raised over $65,000 for The ALS Association, so far.
The ALS community recently presented its recommendations to the U.S. Food and Drug Administration (FDA) regarding the Amyotrophic Lateral Sclerosis: Developing Drugs for Treatment Guidance for Industry at a day-long event, called ALS Community Workshop: Therapy Development and Regulatory Pathways, which was held in Washington, D.C., on July 12. Over 90 people attended in person, with many more tuning in online.