Masitinib may potentially help amyotrophic lateral sclerosis (ALS) patients by targeting mast cells, a Uruguayan study shows. The study, “Evidence for mast cells contributing to neuromuscular pathology in an inherited model of ALS,” appeared in JCI insight. ALS, a neurodegenerative disorder, is characterized by…
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Researchers have created an atlas that links a set of genes in the DNA to a particular trait or disease, a tool that may advance understanding of certain cell characteristics in neurological diseases like Alzheimer’s, Parkinson’s and amyotrophic lateral sclerosis (ALS). This atlas can be further developed and updated by…
Military veterans in the United States who were deployed to World War II or who handled burning agents during their service show shorter amyotrophic lateral sclerosis (ALS) survival, a new study finds. The research, “Military service, deployments, and exposures in relation to amyotrophic lateral sclerosis survival,” appeared in…
More therapies are now available for the 30 million or so people with rare diseases in the U.S. than ever before, and millions of dollars are being invested in clinical studies that will test new ways of evaluating — and advancing — potential treatments, including the use of natural history…
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Columbia Researchers Receive Funding from Facebook Founder to Create Atlas of Spinal Cord Cells
Columbia University medical researchers have received a grant from the Chan Zuckerberg Initiative, named after Facebook founder Mark Zuckerberg and his wife Priscilla Chan, to construct an atlas of gene activity in all cells in the spinal cord. The atlas has the potential to become a reference for investigators researching injuries…
Voyager Therapeutics has recently presented data of its leading adeno-associated virus (AAV) gene therapy for several neurological diseases, including amyotrophic lateral sclerosis (ALS), at the Congress of the European Society of Gene and Cell Therapy (ESGCT), Oct. 17-20 in Berlin, Germany. In their poster, “Translation of Intrathecal Delivery of an AAV Gene…
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#NORDsummit – Despite Criticism, Orphan Drug Act Is Working to Advance Needed Treatments, FDA Says
As Congress begins debate this week to overhaul the U.S. tax code, lawmakers should leave the Orphan Drug Act (ODA) — and the tax incentives it offers pharmaceutical companies to develop therapies for rare diseases — off the table. That’s the message being pushed by the National Organization for…
The presence of diabetes is associated with a 70 percent reduction in the risk of amyotrophic lateral sclerosis (ALS), new study shows. The study, titled “The role of pre-morbid diabetes on developing Amyotrophic Lateral Sclerosis,” was published in the European Journal of Neurology. ALS patients tend…
BrainStorm Cell Therapeutics announced that the first patients have been enrolled in the Phase 3 clinical trial of NurOwn for the treatment of amyotrophic lateral sclerosis (ALS). NurOwn is a mesenchymal stem cell (MSC) platform created for the treatment of ALS. Because it is made from MSCs,…
Ipsen Biopharmaceuticals, an affiliate of Ipsen, announced that five abstracts discussing Dysport (abobotulinumtoxinA) were presented at the American Academy of Physical Medicine and Rehabilitation (AAPM&R) Annual Assembly, held Oct. 12-15 in Denver, Colorado. Dysport, a botulinum toxin product, is approved for treatment of spasticity, or shaking, in adult patients, which…
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