Dec 14, 2022, 07:00 ET AOC 1001 delivered siRNA to skeletal muscle and produced meaningful DMPK reduction in 100% of participants with a 45% mean reduction in DMPK after a single dose of 1 mg/kg or two doses of 2 mg/kg AOC 1001 produced splicing improvement of…
The researchers confirmed that the antiviral effect was linked to PIKfyve rather than an unrelated action of apilimod. Silencing PIKfyve using small interfering RNA reduced HEV infection by approximately 40%, while two additional PIKfyve inhibitors, YM201636 and APY0201, also suppressed infection. The effect…
AASLD The Liver Meeting® 2026 The AX-0810 Phase 1 study will be featured in two poster presentations at The Liver Meeting® 2026, the annual meeting of the American Association for the Study of Liver Diseases (AASLD), taking place November 5–9, 2026. The presentations will focus…
Statistically, within-group change was tested with Wilcoxon signed-rank tests, and between-group comparisons used Mann-Whitney U tests. Adjunct VR was associated with low rates of adverse effects and a treatment course length comparable to the no-VR group. Within the VR cohort,…
Randomized, placebo-controlled study to evaluate the safety, tolerability, and efficacy in a robust, two-part study First-in-class therapeutic targets Alveolar Epithelial Type 2 (AEC2) cells with the aim to halt disease progression and promote repair of fibrotic damage in IPF The ORBIT-IPF is…
Not just for humans: gene therapy to extend longevity is under investigation for dogs, too.Credit: Juliana Yamada/Los Angeles Times/Getty Reference: Proceedings of the National Academy of Sciences paper A newly discovered amphibian species called Gerrothorax maritimus was…
For people living with depression, finding a treatment that actually works can sometimes take several attempts. Antidepressants may not bring enough relief, while switching medicines or combining treatments can involve more waiting, side effects and uncertainty. The findings, published in…
A new review synthesizes the clinical evidence, toxicity profiles and resistance mechanisms behind six FDA-approved antibody-drug conjugates for blood cancers.
A computational study of twenty-four novel pleconaril derivatives identifies molecule 12b as the strongest binder to the enterovirus D68 capsid and delivers validated 3D-QSAR and pharmacophore models for future antiviral design.
Amanita muscaria mushrooms have been used in Siberian shamanism for centuries, or perhaps as long as thousands of years. Some authors have speculated that the mushroom could be associated with other historical and cultural phenomena such as the mysterious magical…
From biological target to tested medicine: target discovery, drug screening and design, clinical trial readouts and psychedelic science.
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