Duchenne muscular dystrophy is a severe and progressive disorder caused by mutations in the dystrophin (DMD) gene that lead to malfunction or absence of dystrophin. This protein stabilizes the sarcolemma and protects muscle cells during contraction.
Shiratronics Inc. raised $66 million in an oversubscribed series B financing round to support completion of its recently launched clinical trial to evaluate the safety and efficacy of its neuromodulation device in patients with treatment-resistant chronic migraine.
Atai Therapeutics Inc. has identified 3-alkylamine indole compounds acting as 5-HT2A receptor agonists and reported to be potentially useful for the treatment of anxiety, eating and neurological disorders.
Miralinc Pharma Inc. has patented new heterocyclic selective histone deacetylase 6 (HDAC6) inhibitors reported to be useful for the treatment of type 2 Charcot-Marie-Tooth disease and chemotherapy-induced peripheral neuropathy, among others.
Humanwell Pharmaceutical US Inc. has synthesized new sigma non-opioid intracellular receptor 1 (SIGMAR1) antagonists reported to be useful for the treatment of cancer, pain, psychosis and substance abuse and dependence.
The central nervous system (CNS) in adult mammals has limited regeneration capacity, and traumatic injuries to the CNS usually lead to permanent functional impairment.
Centessa Pharmaceuticals plc recently detailed the discovery and preclinical characterization of a novel potent and selective orexin OX2 receptor agonist, ORX-142.
Bad news has buffeted Sage Therapeutics Inc. twice in the past few months. Now its placebo-controlled phase II Lightwave study of dalzanemdor in Alzheimer's disease has missed the primary outcome measure, prompting the company to stop development of the NMDA receptor positive allosteric modulator in the indication.
Aviadobio Ltd. has entered a potential $2.18 billion license and commercialization agreement for its frontotemporal dementia gene therapy, AVB-101, with Astellas Pharma Inc. Astellas is making a $20 million equity investment in London-based Aviadobio and will pay up to $30 million up front in advance of deciding whether or not to exercise the exclusive option to worldwide rights.
A digital cognitive assessment developed by Braincheck Inc., Braincheck Assess, proved comparable to the gold-standard Montreal Cognitive Assessment in identifying patients with cognitive impairment in a study published in Frontiers in Psychology.
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