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Home » Topics » BioWorld Science, Neurology/psychiatric

BioWorld Science, Neurology/psychiatric
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Neurology/psychiatric

Dewpoint Therapeutics patents new TARDBP modulators

July 25, 2025
Dewpoint Therapeutics Inc. has described TAR DNA-binding protein 43 (TARDBP; TDP-43) modulators reported to be useful for the treatment of traumatic brain injury, frontotemporal dementia and amyotrophic lateral sclerosis.
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Stroke illustration: brain, artery, neurons
Neurology/psychiatric

Idebenone derivative shows promise against ischemic stroke

July 25, 2025
No Comments
Stroke is the third leading cause of disability worldwide, and its incidence is expected to increase as the global population ages. Idebenone can promote recovery after stroke, but it is less effective during the acute phase of stroke.
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Parkinson's disease illustration showing neurons containing alpha-synuclein
Neurology/psychiatric

MJFF grant to support Congruence Therapeutics’ GBA1 Parkinson’s disease efforts

July 25, 2025
No Comments
Congruence Therapeutics Inc. has received a research grant of $5 million from The Michael J. Fox Foundation for Parkinson's Research (MJFF) to advance its GCase-targeting small molecules for GBA1 Parkinson’s disease. Mutations of the GBA1 gene, encoding the enzyme GCase, represent the single largest genetic risk factor for Parkinson’s disease.
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Illustration of Microglia cells (red) in Alzheimer´s disease
Neurology/psychiatric

Two cancer drugs combined modify Alzheimer’s disease in mice

July 25, 2025
By Mar de Miguel
No Comments
Current treatments for Alzheimer’s disease have limited effects. While they can slow cognitive decline or alleviate symptoms, they do not reverse this complex neurodegenerative condition caused by multiple factors. Researchers from the Gladstone Institutes and the University of California, San Francisco (UCSF) have screened FDA-approved drugs in search of agents that could potentially modify the disease.
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Neurology/psychiatric

TREM mediates neuroinflammation and synaptic dysfunction in TBI

July 24, 2025
No Comments
Researchers from Peking University and the China-Japan Friendship Hospital have gained new insights into the role of triggering receptor expressed on myeloid cells-1 (TREM-1) in traumatic brain injury (TBI). Cerebral damage resulting from external mechanical impacts causes TBI, which is a leading cause of morbidity and mortality worldwide.
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Hands holding brain
Neurology/psychiatric

PX5-9, an optimized cinnamanilide derivative with neuroprotective potential

July 23, 2025
No Comments
Brain-derived neurotrophic factor (BDNF) is a neurotrophin that supports neuronal growth and survival and plays a critical role in regulating synaptic plasticity and cognitive function. Reduced BDNF levels have been observed in Alzheimer’s disease, Parkinson’s disease and mood disorders, suggesting that agents capable of enhancing BDNF expression may represent promising therapeutic strategies for both neurodegenerative diseases and depression.
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Doctor examining child in wheelchair
Neurology/psychiatric

Precision Biosciences’ PBGENE-DMD gains US orphan drug designation

July 23, 2025
No Comments
The U.S. FDA has granted Precision Biosciences Inc.’s PBGENE-DMD orphan drug designation for the treatment of Duchenne muscular dystrophy (DMD). PBGENE-DMD uses two complementary Arcus nucleases delivered via a one-time administration in a single AAV to excise exons 45-55 of the dystrophin gene in order to restore near full-length dystrophin protein within the body to improve functional outcomes.
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Neurology/psychiatric

GABRA5 modulation supports treatment of neurodevelopmental disorders

July 23, 2025
No Comments
Alterations in the GABAergic pathway in the brain contribute to the pathophysiology of neurodevelopmental disorders, including autism spectrum disorder and Angelman syndrome. Targeting the GABA-A receptor α5 subunit (GABRA5) with positive allosteric modulators represents a new therapeutic strategy for the treatment of these conditions.
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Neurology/psychiatric

CSPC Zhongnuo Pharmaceutical identifies new MAO-B inhibitors

July 22, 2025
CSPC Zhongnuo Pharmaceutical (Shijiazhuang) Co. Ltd. has reported new monoamine oxidase-B (MAO-B) inhibitors potentially useful for the treatment of depression, stroke, hypertension, obesity, Alzheimer’s and Parkinson’s disease.
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Illustration of DNA double helix and motorized wheel chair
Neurology/psychiatric

cGMP-compliant iPSC-based myogenic progenitor therapy shows preclinical promise for DMD

July 22, 2025
No Comments
Researchers at the University of Minnesota and collaborating institutions have developed a promising stem cell-based therapy for the treatment of muscular dystrophies. The team has successfully created a novel myogenic progenitor cell product called Myopaxon, derived from human-induced pluripotent stem cells (iPSCs).
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