Neuropathy symptom and change: Inotersen treatment of hereditary transthyretin amyloidosis.,Muscle & Nerve

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Neuropathy symptom and change: Inotersen treatment of hereditary transthyretin amyloidosis.
Muscle & Nerve ( IF 2.8 ) Pub Date : 2020-07-12 , DOI: 10.1002/mus.27023
P James B Dyck ₁ , Teresa Coelho ₂ , Marcia Waddington Cruz ₃ , Thomas H Brannagan ₄ , Sami Khella ₅ , Chafic Karam ₆ , John L Berk ₇ , Michael J Polydefkis ₈ , John C Kincaid ₉ , Janice F Wiesman ₁₀ , William J Litchy ₁ , Michelle L Mauermann ₁ , Elizabeth J Ackermann ₁₁ , Brenda F Baker ₁₂ , Shiangtung W Jung ₁₂ , Spencer Guthrie ₁₃ , Michael Pollock ₁₄ , Peter J Dyck ₁

Affiliation

Department of Neurology, Mayo Clinic, Rochester, Minnesota, USA.
Department of Neurosciences, Centro Hospitalar do Porto, Porto, Portugal.
Department of Neurology, Federal University of Rio de Janeiro, University Hospital, Rio de Janeiro, Brazil.
Department of Peripheral Neuropathy, Columbia University Medical Center, New York, New York, USA.
Department of Neurology, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
Department of Neurology, Oregon Health & Science University, Portland, Oregon, USA.
Department of Medicine, Boston University, Boston, Massachusetts, USA.
Department of Neurology, Johns Hopkins University, Baltimore, Maryland, USA.
Department of Neurology, Indiana University, Indianapolis, Indiana, USA.
Department of Neurology, New York University, New York, New York, USA.
Otonomy, Inc, San Diego, California, USA.
Ionis Pharmaceuticals, Inc., Carlsbad, California, USA.
Aurora Bio, San Francisco, California, USA.
Akcea Therapeutics, Inc., Boston, Massachusetts, USA.

Hereditary transthyretin‐mediated amyloidosis (hATTR) manifests as multisystem dysfunction, including progressive polyneuropathy. Inotersen, an antisense oligonucleotide, improved the course of neuropathic impairment in patients with hATTR in the pivotal NEURO‐TTR study (NCT01737398). To determine inotersen's impact on symptoms and patients’ neuropathy experience, we performed a post hoc analysis of the Neuropathy Symptoms and Change (NSC) score.

中文翻译：

神经病症状和变化：伊诺特森治疗遗传性甲状腺素转运蛋白淀粉样变性。

遗传性甲状腺素介导的淀粉样变性病（hATTR）表现为多系统功能障碍，包括进行性多发性神经病。在关键的NEURO-TTR研究（NCT01737398）中，Inotersen是一种反义寡核苷酸，可改善hATTR患者的神经性病变进程。为了确定inotersen对症状和患者神经病经验的影响，我们对神经病症状和变化（NSC）评分进行了事后分析。

更新日期：2020-09-20

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