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Radicava and Its Swift FDA Approval Are Reasons for Hope, ALS Association Official Says
Radicava (edaravone), the first approved treatment for amyotrophic lateral sclerosis in 22 years, is being welcomed with “a great deal of excitement” in the ALS community, an ALS Association executive said. The excitement stems both from Radicava’s potential to treat the disease and the hope that its approval will lead to future treatment authorizations, said Calaneet Balas, the […]
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Transcript of Interview with ALS Association’s Calaneet Balas on Radicava, the FDA, Insurance and Pricing
Below is a transcript of the ALS News Today interview with Calaneet Balas, executive vice president of strategy for the ALS Association. The nonprofit association, established in 1985, is active in promoting research into ALS, providing assistance to patients through its network of 39 state chapters, and in coordinating care though its certified ALS clinical care centers, now […]
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Edaravone, Now Known as Radicava, Has Been ‘Minor Miracle’ for Some, ALS Worldwide Co-director Says
Edaravone — now more widely known by its U.S. brand name, Radicava — has produced “a minor miracle” for some amyotrophic lateral sclerosis patients, said Stephen Byer, co-director of ALS Worldwide. “We have found and continue to find great benefit in terms of slowing, stopping and, in some cases, even helping to mildly reverse some […]
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New Technique Gives Researchers a Near-atom-level View of Enzyme’s Role in Correcting Misfolded Proteins
University of Michigan and University of Pennsylvania researchers used a cutting-edge technique called cryo-electron microscopy to see how the Hsp104 yeast enzyme breaks up clumps of the misfolded proteins that are hallmarks of neurodegenerative diseases like ALS. The resolution that the technique provided was so high that it was close to the level of individual atoms. Researchers discovered that Hsp104 […]
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Avoid Mental Potholes While Navigating Your Life
In my last column, I shared how I’m always on alert for speed bumps as I travel down life’s highway, while living with ALS. These are the metaphorical unknowns that pop up, and challenge my resilience and positive mindset. But in addition to speed bumps, it’s the mental potholes that I really try to avoid. We […]
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Mutation That Interferes with Genetic Instructions May Cause Hereditary ALS, Study Reports
Harvard Medical School researchers have discovered how a mutation of the C9ORF72 gene causes the changes in energy production and nerve cell function that are linked to inherited forms of ALS and frontotemporal dementia, or FTD. The mutation produces two toxic proteins that can disrupt the normal processing of genetic instructions, the team said. Researchers published […]
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Level of Neurofilaments in Cerebrospinal Fluid Could Confirm ALS Diagnosis, Study Reports
Measuring neurofilaments in brain and spinal fluid appears to be a reliable way of confirming an ALS diagnosis, Belgian and German researchers report. This finding is important because it takes an average of a year from the appearance of the first symptoms before doctors can diagnose the disease. The discovery could shorten this time. The neurons […]
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Israel’s BrainStorm Receives $2.1 Million Grant to Support Development of Stem-Cell Therapy NurOwn to Treat ALS
The Israel Innovation Authority (IIA) has awarded BrainStorm Cell Therapeutics a $2.1 million grant to support the development of NurOwn, an innovative mesenchymal stem cell-based platform to treat neurodegenerative diseases including amyotrophic lateral sclerosis (ALS). NurOwn was developed by Tel Aviv University professors Dani Offen and the late Eldad Melamed. NurOwn builds on research developing mesenchymal stem cells […]
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Metal Levels in Blood, Urine, and Hair May Serve as ALS Biomarkers, Study Suggests
Several metals may be linked to the risk of amyotrophic lateral sclerosis (ALS), according to results of a small study in Italy. The findings state that levels of metals and other essential trace elements in blood, urine, and hair may serve as biomarkers to diagnose and monitor ALS. The study titled, “Essential trace elements in amyotrophic […]
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Onetime Malaria Therapy Pyrimethamine Reduces Levels of ALS-linked Protein, Study Finds
The former malaria therapy pyrimethamine reduces levels of an ALS-linked protein in the spinal fluid of patients whose disease stems from gene mutations, a Phase 1/2 clinical trial shows. The protein is SOD1. The mutations connected with amyotrophic lateral sclerosis are in the SOD1 gene that controls the protein. The study, “Pyrimethamine significantly lowers cerebrospinal […]
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