Researchers generated motor neurons from amyotrophic lateral sclerosis (ALS) patients with a mutated gene found in familial forms of the disease. The study sheds new light on the disease mechanism. Transport of materials up and down the motors’ neuron length (axon) keeps nerve cell messages…
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Relatives of patients with amyotrophic lateral sclerosis (ALS) have a higher likelihood of developing neuropsychiatric symptoms including schizophrenia, obsessive-compulsive disorder, autism, and alcoholism, research from Ireland shows. The study, “Clustering of Neuropsychiatric Disease in First-Degree and Second-Degree Relatives of Patients With Amyotrophic Lateral Sclerosis,” was published…
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…
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…
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