AveXis, which is running clinical trials of a promising gene therapy for spinal muscular atrophy (SMA) — and developing a potentially similar therapy for a genetic form of amyotrophic lateral sclerosis (ALS) — announced plans to be acquired by Novartis for $8.7 billion in cash. AVXS-101 is the…

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…

Inhibition of a key protein called dual leucine zipper kinase (DLK), a neuronal injury sensor, is protective in mice with amyotrophic lateral sclerosis (ALS) and Alzheimer’s disease and shows potential for future therapeutic strategies, according to researchers. The study, “Loss of dual leucine zipper kinase signaling…

AveXis and REGENXBIO have signed a new licensing agreement to develop therapies for a particular form of ALS and for Rett syndrome. They already have an agreement that covers spinal muscular atrophy therapies. The form of amyotrophic lateral sclerosis that the new agreement covers is the one caused by mutations…

Preclinical testing will soon begin in VY-SOD101, a compound seen as a potential treatment for a certain gene mutation found in people with familial amyotrophic lateral sclerosis (ALS).  Its developer,  Voyager Therapeutics, hopes to follow this work, if successful, with clinical trials in patients  in about two years. Patients with ALS typically develop weakness…

Measuring copper concentration in the blood may allow for the diagnosis of amyotrophic lateral sclerosis (ALS) before the onset of clinical symptoms, according to new research in mice. The study, developed by researchers at the University of Wollongong in Australia, might provide a new means for ALS diagnosis that does not require disease…

Researchers at the Karolinska Institute in Sweden and the University of Barcelona in Spain studied the contribution of cellular redox environment changes to the stability and integrity of SOD1, a protein implicated in the development and progression of familial amyotrophic lateral sclerosis (ALS). A key finding was that mutations in the…

https://www.youtube.com/watch?v=ZU-wsH2do8Q In this video by Neurology Today learn more about neuroimaging. Individuals with mutations in the superoxide dismutase 1 (SOD1) gene develop brain white matter abnormalities before the onset of symptoms of amyotrophic lateral sclerosis (ALS), according to a new imaging study that was presented at the 2014 AAN annual…

ISIS Pharmaceuticals, in collaboration with Biogen, has started a Phase 1/2 clinical study of ISIS-SOD1Rx (BIIB067), one of the drug candidates to come out of the strategic partnership between the two companies, in adult patients diagnosed with amyotrophic lateral sclerosis (ALS). ISIS-SOD1Rx’s mode of action relies on ISIS antisense technology that targets at…

Researchers from the University of Milan (UM), Italy, have recently released findings from their scientific literature review, in an effort to uncover the causes of cellular pathologies that are the hallmark of Amyotrophic Lateral Sclerosis (ALS). The findings entitled, “SOD1 misplacing and mitochondrial dysfunction in amyotrophic lateral sclerosis pathogenesis“, published…