Skyhawk Therapeutics and Celgene have agreed to collaborate for five years to discover, develop, and commercialize innovative small molecule treatment options for neurological diseases with high unmet medical needs, including amyotrophic lateral sclerosis (ALS) and Huntington’s disease. Several genetic disorders are caused by “exon skipping,” which means…
Rare gene mutations in amyotrophic lateral sclerosis (ALS) will be increasingly identified with large-scale, international studies, findings from Project MinE suggest. The study, “Project MinE: study design and pilot analyses of a large-scale whole-genome sequencing study in amyotrophic lateral sclerosis,” was published in the European…
NurOwn, an amyotrophic lateral sclerosis (ALS) treatment candidate, will not be made available under the new Right to Try Act at this time due to a lack of funding alternatives for patients, BrainStorm Cell Therapeutics announced in a press release. The company said it reached this decision after carefully evaluating the Right to…
ProMIS Neurosciences announced that it is moving toward selecting antibodies — from the many identified in earlier work — that specifically target the toxic proteins that accumulate in the brains of amyotrophic lateral sclerosis (ALS) and Alzheimer’s patients, and that may work as therapies. Specifically for ALS, the identified antibodies target TDP43…
Determining genetic profiles from patients’ blood samples can provide a broad view of the biological processes involved in amyotrophic lateral sclerosis (ALS). However, this information may not add valuable diagnostic information because it can’t accurately discriminate ALS cases from conditions that mimic the disease, according to researchers. Their study,…
The noninvasive detection of changes in the blood specific to amyotrophic lateral sclerosis (ALS) may improve the diagnosis of the disease, according to researchers. Their study, “Initial Identification of a Blood-Based Chromosome Conformation Signature for Aiding in the Diagnosis of Amyotrophic Lateral Sclerosis,” appeared in the journal…
A recent study highlights a previously unknown function of the TDP-43 protein in nerve cells that may contribute to the progression of amyotrophic lateral sclerosis (ALS). The finding adds a new layer of knowledge that could help to better understand the mechanisms involved in ALS development and progression. TDP-43 protein…
The National Institute of Neurological Disorders and Stroke (NINDS) has awarded Aquinnah Pharmaceuticals $3.4 million to continue to develop and advance potential therapies to treat amyotrophic lateral sclerosis (ALS). This NINDS grant is the second given to Aquinnah to help support its novel approach for the treatment of…
Online health forums offers patients with chronic diseases a way to share experiences and develop connections with others in a similar situation — gaining access to a community that can provide both empathy and support, a study shows. How empathy might develop in online exchanges was the focus of the…
The National Institute of Neurological Disorders and Stroke (NINDS) awarded $3.7 million to AcuraStem to support the development of a small molecule drug to treat C9orf72 gene-related amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). The Small Business Innovation Research (SBIR) fast-track grant (R44NS105156) will promote the development…
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