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BioWorld - Wednesday, May 31, 2023
Home » Topics » Disease categories and therapies » Neurology/Psychiatric

Neurology/Psychiatric
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Muscular dystrophy
Neurology/Psychiatric

Researchers create new mouse model of DMD using knock-in of human exon 50

May 26, 2023
No Comments
Researchers from HuidaGene Therapeutics Co. Ltd. have evaluated the effects of adenine base editing (ABE)-induced exon skipping of exon 50 in a humanized mouse model of Duchenne muscular dystrophy (DMD).
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Endocrine/Metabolic

AAV9-CLN5 improves symptoms in mice with Batten disease

May 26, 2023
No Comments
Neuronal ceroid lipofuscinosis, commonly known as Batten disease, is an inherited pediatric neurodegenerative lysosomal storage disease caused by mutations in the CLN5 gene. The disease is incurable and there is an urgent medical need for novel therapies. A murine model of Batten disease was developed to study a novel AAV vector that expresses CLN5, AAV9-CLN5. In the study by University College London investigators, the gene therapy, driven by the synapsin promoter, was intracerebroventricularly administered into neonatal Cln5-knockout mice.
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Neurology/Psychiatric

MyoDys45-55 restores dystrophin and improves functional measures in murine model of DMD

May 26, 2023
No Comments
Myogene Bio LLC has developed an approach through CRISPR7Cas9 technology...
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Man sitting on bed holding head
Neurology/Psychiatric

Psilera nominates non-hallucinogenic psilocybin derivative as lead clinical candidate

May 26, 2023
No Comments
Psilera Inc. has selected a novel, non-hallucinogenic psilocybin derivative, PSIL-006...
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Brain and neural networks
Neurology/Psychiatric

Hanall and Daewoong partner with Nurron to advance compounds for neurodegenerative disorders

May 26, 2023
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Hanall Biopharma Co. Ltd. and Daewoong Pharmaceutical Co. Ltd. have entered into...
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Endosome
Neurology/Psychiatric

Experimental painkiller blocks GPCR signaling inside the cell

May 26, 2023
By Subhasree Nag
No Comments
Researchers have developed a new highly effective therapeutic for pain relief by altering the chemical properties of an antinausea drug, netupitant. The modified drug is able to enter the intracellular membrane-bound endosome and target the GPCRs therein, rather than at the cell surface, that leads to optimal pain relief. The study, published in the Proceedings of the National Academy of Sciences (PNAS) on May 22, 2023, was led by Nigel Bunnett, Professor and Chair of Molecular Pathobiology at NYU College of Dentistry, and illustrates how GPCR-mediated pain signaling occurs inside the endosomes rather than at the surface, highlighting the need for drugs that can reach receptors within the cells itself.
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Bioxcel has mixed phase III data and tough stock day

May 25, 2023
By Lee Landenberger
No Comments
Mixed phase III study results have Bioxcel Therapeutics Inc. moving ahead to complete the clinical trial while withstanding a hammering from investors. BXCL-501 (dexmedetomidine), a sublingual film being developed to treat bipolar disorders- or schizophrenia-associated agitation, produced clinically meaningful efficacy results in part 1 of the pivotal study with half of the approved dose, but the primary efficacy endpoint was not statistically significant at two hours (p=0.077). BXCL-501 separated from placebo at four hours (p=0.049).
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onward.jpg

Let there be walk: Onward Medical sees first use of movement-restoring lead

May 25, 2023
By Nuala Moran
No Comments
The researchers who enabled patients with spinal cord injuries to walk independently after implanting programmable electrodes below their lesions have now taken things one step further, restoring direct communication from the brain to the spinal cord, enabling the brain rather than an external computer to direct leg movements.
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onward.jpg
Neurology/Psychiatric

Let there be walk: Onward Medical sees first use of movement-restoring lead

May 25, 2023
By Nuala Moran
No Comments
The researchers who enabled patients with spinal cord injuries to walk independently after implanting programmable electrodes below their lesions have now taken things one step further, restoring direct communication from the brain to the spinal cord, enabling the brain rather than an external computer to direct leg movements.
Read More
Neurology/Psychiatric

Marvel Biotechnology patent details new compounds for treatment of depression

May 25, 2023
No Comments
Marvel Biotechnology Inc. has reported the identification of compounds potentially useful for the treatment of depression.
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