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Spinal muscular atrophy: A test case for drug development in orphan diseases

机译：脊髓性肌萎缩症：孤儿疾病药物开发的测试案例

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期刊名称 NeuroRx
作者
Lisa J. Bain;
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作者单位

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年(卷),期 2006(3),4
年度 2006
页码 535–539
总页数 5
原文格式 PDF
正文语种
中图分类神经科学;
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1. Spinal muscular atrophy: A test case for drug development in orphan diseases [J] . Lisa J. Bain Neurotherapeutics . 2006,第4期

机译：脊髓性肌萎缩症：孤儿疾病药物开发的测试案例
2. Nucleic Acid–Targeted Small Molecules have Therapeutic Potential in the Treatment of Spinal Muscular Atrophy: Small‐molecule drugs that can selectively bind RNA and modulate pre‐mRNA splicing have potential as a treatment strategy for human disease, including spinal muscular atrophy [J] . American journal of medical genetics, Part A . 2018,第8期

机译：核酸靶心的小分子在治疗脊柱肌萎缩中具有治疗潜力：可以选择性地结合RNA并调节前mRNA剪接的小分子药物作为人类疾病的治疗策略，包括脊柱肌肉萎缩
3. Anaesthesia and orphan disease: a patient with spinal muscular atrophy type III (Wohlfart - Kugelberg - Welander syndrome) undergoing laparoscopic cholecystectomy [J] . El Harchaoui A. Karim, Dwars Boudewijn J., Soesan Marcel, European journal of anaesthesiology . 2015,第3期

机译：麻醉和孤儿疾病：接受腹腔镜胆囊切除术的III型脊髓性肌萎缩症（Wohlfart-Kugelberg-Welander综合征）患者
4. Robotic stretcher for spinal muscular atrophy patient: Preliminary tests of user controllability [C] . Sakaki Taisuke, Aoki Kanta, Ushijima Kuniharu, International Conference on Ubiquitous Robots and Ambient Intelligence . 2013

机译：脊髓性肌萎缩症患者的机器人担架：用户可控性的初步测试
5. Glycyl-tRNA synthetase mutations cause Charcot-Marie-Tooth disease type 2D and distal spinal muscular atrophy type V: A potentially novel disease mechanism for human peripheral neuropathies. [D] . Antonellis, Anthony. 2005

机译：糖基-tRNA合成酶突变导致2D型Charcot-Marie-Tooth病和V型远端脊髓性肌萎缩症：人类周围神经病的潜在新型疾病机制。
6. Spinal Muscular Atrophy: The Development and Implementation of Potential Treatments Running Head: Spinal Muscular Atrophy [O] . W. David Arnold, Arthur H.M. Burghes -1

机译：脊髓性肌萎缩症：潜在治疗方法的开发和实施
7. Anaesthesia and orphan disease: a patient with spinal muscular atrophy type III (Wohlfart-Kugelberg-Welander syndrome) undergoing laparoscopic cholecystectomy [O] . El Harchaoui A. Karim, Dwars Boudewijn J., Soesan Marcel, 2015

机译：麻醉和孤儿疾病：腹腔镜胆囊切除术的三型脊髓性肌萎缩症（Wohlfart-Kugelberg-Welander综合征）患者

Spinal muscular atrophy: A test case for drug development in orphan diseases

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