Success by nearly all measures with Praxis Precision Medicines Inc.’s essential tremor phase III candidate, ulixacaltamide, drove the company’s stock up significantly and provided solid data for an NDA filing planned for early 2026.
At the 2025 European College of Neuropsychopharmacology (ECNP) Congress in Amsterdam this week, researchers presented new findings on the role of endocannabinoids in stress, addiction … and the integrity of the blood-brain-barrier.
Researchers at Indian Institute of Technology (BHU) Varanasi and collaborators have designed inhibitors of cholinesterase based on benzothiazole-phenylpiperizine as a potential treatment for Alzheimer’s disease.
Researchers at Shanghai Jingxin Biopharmaceutical Co. Ltd. and Zhejiang Jingxin Pharmaceutical Co. Ltd. have divulged compounds acting as leucine-rich repeat kinase 2 (LRRK2; dardarin) inhibitors reported to be useful for the treatment of Parkinson’s disease.
Essential tremor is a movement disorder marked by involuntary, rhythmic shaking in the hands but sometimes affecting the head, voice and other areas. Its exact cause is still unknown. Recent research suggests that dysfunction in the neurotransmitter systems, particularly involving γ-aminobutyric acid (GABA), contributes significantly to the disorder.
Neurocrine Biosciences Inc. has disclosed vesicular monoamine transporter 2 (VMAT2) inhibitors reported to be useful for the treatment of neurological and psychiatric disorders, among others.
Some 1 million people around the world suffer severe neurological problems, such as epilepsy, motor impairment and cognitive dysfunction, because they express insufficient SynGAP1, a GTPase-activating protein that operates postsynaptically. Current therapies can mitigate symptoms but not cure the underlying disease. Researchers at the Allen Institute and collaborators have demonstrated a potential genetic cure for SynGAP1 deficiency.
Tr1x Inc. has obtained IND clearance from the FDA for TRX-319, paving the way for initiation of a phase I/IIa study in progressive multiple sclerosis (MS) early next year. TRX-319 is designed to combine targeted B-cell control with active anti-inflammatory signaling and T-cell regulation, with the goal of restoring immune balance.
Scientists at Indiana University and Purdue Research Foundation have synthesized inositol polyphosphate-5-phosphatase D (SHIP-1; INPP5D) inhibitors reported to be useful for the treatment of mild cognitive impairment, Alzheimer’s disease, vascular cognitive impairment, Lewy body dementia and frontotemporal dementia.
Digital mental health tools are popping up with some regularity lately, but both Australia’s Therapeutic Goods Administration and the U.S. FDA have enough concerns about these products that they are taking a closer look at their risks and benefits.
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