FDA approves QALSODY™ (tofersen) targeting a genetic cause of ALS

 25 April 2023: Ionis Pharmaceuticals, Inc today announced that its partner Biogen has received U.S. Food and Drug Administration (FDA) approval of QALSODY™ (tofersen) 100 mg/15mL injection for the treatment of amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene. This indication is approved under accelerated approval…

Gene in the brain can reduce anxiety in animal model

A gene in the brain driving anxiety symptoms has been identified by an international team of scientists. Modification of the gene is shown to reduce anxiety levels, offering a novel drug target for anxiety disorders. The discovery, led by researchers at the Universities of Bristol and Exeter, was published online online on 25th April in Nature…

#AAN2023: Atogepant for preventive treatment of episodic migraine

21 April 2023: AbbVie has announced positive data from its Phase 3 ELEVATE study, evaluating atogepant for the preventive treatment of episodic migraine in people who had previously failed two to four classes of oral preventive medications. The results of the study demonstrated adult patients in the atogepant 60 mg once daily (QD) arm experienced…

Study finds Pison AI technology can detect neurological disorder in ALS patients

Lewis Katz School of Medicine at Temple University research shows Pison’s Neural Biosensor can potentially enable remote monitoring, early detection and intervention for neurological diseases Pison have announced the results of recent clinical trial conducted by researchers at the Lewis Katz School of Medicine at Temple University’s MDA/ALS Center of Hope, in the US. The trials concluded…

Why is Alzheimer’s Gene APOE4 less dangerous for people of African descent?

Researchers at the John P Hussman Institute for Human Genomics at the University of Miami Miller School of Medicine, US have found that variations in chromatin accessibility, and thus gene expression, may explain why people of European descent with APOE4 gene variants have a greater risk of developing Alzheimer’s disease than people of African descent with similar…

Powerful AI algorithm to gain new insights into deadly glioblastoma

Scientists at Sylvester Comprehensive Cancer Center at the University of Miami Miller School of Medicine, US collaborating with international researchers, have developed a sophisticated AI algorithm that performs advanced computational analysis to identify potential therapeutic targets for glioblastoma multiforme (GBM) and other cancers. Their research is described in the Feb. 2 issue of the journal…

Rozanolixizumab and Zilucoplan Phase 3 Generalised Myasthenia Gravis studies

13 April 2023: UCB, a global biopharmaceutical company, announced that The Lancet Neurology has published data from the Phase 3 MycarinG study evaluating the efficacy and safety of rozanolixizumab in adult patients with acetylcholine receptor autoantibody-positive (AChR-Ab+) or muscle-specific tyrosine kinase autoantibody-positive (MuSK-Ab+) generalised myasthenia gravis (gMG) and the Phase 3 RAISE study evaluating the…

Drug to lower brain pressure could treat blinding IIH headaches

March 13, 2023: Patients with ‘blinding’ headaches known as Idiopathic Intercranial Hypertension (IIH) could be treated with an injectable peptide used for type 2 diabetes, a new trial has found. The study, published in the journal Brain, reports on a phase two trial of a drug called exenatide, a GLP-1 receptor agonist, as a potential…