Ontology highlight
ABSTRACT:
SUBMITTER: Dyck PJB
PROVIDER: S-EPMC7540369 | biostudies-literature | 2020 Oct
REPOSITORIES: biostudies-literature
Dyck P James B PJB Coelho Teresa T Waddington Cruz Marcia M Brannagan Thomas H TH Khella Sami S Karam Chafic C Berk John L JL Polydefkis Michael J MJ Kincaid John C JC Wiesman Janice F JF Litchy William J WJ Mauermann Michelle L ML Ackermann Elizabeth J EJ Baker Brenda F BF Jung Shiangtung W SW Guthrie Spencer S Pollock Michael M Dyck Peter J PJ
Muscle & nerve 20200807 4
<h4>Introduction</h4>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.<h4>Methods</h4>Stage 1 or 2 ...[more]