When discussing potential Radicava (edaravone) treatment with amyotrophic lateral sclerosis (ALS) patients, physicians need to take great care in presenting facts in an ethical and scientifically sound manner, warn two researchers who believe that mainstream reports of the treatment may be misleading. The duo — Crystal Yeo, MD, PhD,…
A new model for predicting ALS patients’ survival looks promising, University of Michigan researchers report. In fact, it want a competition for models that can forecast survival. A combination of patients’ ability to walk, breathe and eat, plus their scores on a physical functioning scale and a lung function measure…
A traffic jam inside nerve cells is a feature of most forms of amyotrophic lateral sclerosis and frontotemporal dementia, a study shows. It suggests that easing the jam is a potential strategy for treating ALS. Therapies to do this are moving into clinical trials, the researchers said. The study, “…
A protein called neuronal apoptosis inhibitor protein (NAIP) is a potential disease marker and predictor of outcomes in amyotrophic lateral sclerosis (ALS), according to a new study. The protein’s levels in ALS patients and controls were measured in blood samples in the study. The…
A dissolving tablet version of riluzole called BHV-0223 has shown positive bioequivalence when compared to the oral form of the therapy, Rilutek, developer Biohaven Pharmaceuticals announced. The dissolving tablet, placed under the tongue, is easier to administrate to patients with amyotrophic lateral sclerosis (ALS) who have trouble swallowing. BHV-0223 also was found…
NeuroSGC, a new scientific partnership formed by the Structural Genomics Consortium (SGC) and the Montreal Neurological Institute and Hospital (The Neuro) aims to discover new treatments for amyotrophic lateral sclerosis (ALS) and Parkinson’s disease — both of which currently lack effective therapies. Drug discovery for these and other neurological…
The U.S. Food and Drug Administration (FDA) has issued a warning to the American CryoStem Corporation of Monmouth Junction, New Jersey, for marketing Atcell, the company’s adipose-derived stem cell product, without FDA approval. In its warning letter, the FDA noted “significant deviations from current good manufacturing practice requirements, including some…
Researchers at the University of Calgary in Canada are enrolling amyotrophic lateral sclerosis (ALS) patients for a Phase 2 clinical trial to evaluate the potential therapeutics effects of a well-established antipsychotic drug called Orap (pimozide). The research team, led by Dr. Lawrence Korngut, associate professor at the…
Sangamo Therapeutics and Pfizer will work together on gene therapies they hope will overcome mutations that cause ALS and frontotemporal lobar degeneration. Gene therapy works by delivering a healthy copy of a malfunctioning gene to a patient. The therapy that Sangamo is developing replaces a faulty C9orf72 gene. It consists…
Cell stress, such as a viral infection, may trigger the production of faulty proteins from the C9orf72 gene, a leading contributor of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), say researchers at the University of Michigan. Their study, “RAN translation at C9orf72-associated repeat expansions is selectively enhanced…
