新型杂环化合物作为SMN2调节剂通过调节SMN2剪接治疗脊髓性肌萎缩。

IF 3.5 3区医学 Q2 CHEMISTRY, MEDICINAL

ACS Medicinal Chemistry Letters Pub Date : 2025-06-30 eCollection Date: 2025-07-10 DOI:10.1021/acsmedchemlett.5c00364

Ram W Sabnis

引用次数: 0

摘要

本文提供作为SMN2调节剂的新型杂环化合物、药物组合物、通过调节SMN2剪接治疗脊髓性肌萎缩症（SMA）的此类化合物的用途，以及制备此类化合物的方法。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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Novel Heterocyclic Compounds as SMN2 Modulators for Treating Spinal Muscular Atrophy Via Modulation of SMN2 Splicing.

Provided herein are novel heterocyclic compounds as SMN2 modulators, pharmaceutical compositions, use of such compounds in treating spinal muscular atrophy (SMA) via modulation of SMN2 splicing, and processes for preparing such compounds.

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来源期刊

ACS Medicinal Chemistry Letters CHEMISTRY, MEDICINAL-

CiteScore

7.30

自引率

2.40%

发文量

328

审稿时长

1 months

期刊介绍： ACS Medicinal Chemistry Letters is interested in receiving manuscripts that discuss various aspects of medicinal chemistry. The journal will publish studies that pertain to a broad range of subject matter, including compound design and optimization, biological evaluation, drug delivery, imaging agents, and pharmacology of both small and large bioactive molecules. Specific areas include but are not limited to: Identification, synthesis, and optimization of lead biologically active molecules and drugs (small molecules and biologics) Biological characterization of new molecular entities in the context of drug discovery Computational, cheminformatics, and structural studies for the identification or SAR analysis of bioactive molecules, ligands and their targets, etc. Novel and improved methodologies, including radiation biochemistry, with broad application to medicinal chemistry Discovery technologies for biologically active molecules from both synthetic and natural (plant and other) sources Pharmacokinetic/pharmacodynamic studies that address mechanisms underlying drug disposition and response Pharmacogenetic and pharmacogenomic studies used to enhance drug design and the translation of medicinal chemistry into the clinic Mechanistic drug metabolism and regulation of metabolic enzyme gene expression Chemistry patents relevant to the medicinal chemistry field.