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Neuropathy symptom and change: Inotersen treatment of hereditary transthyretin amyloidosis

INTRODUCTION: 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)....

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Bibliografische gegevens
Gepubliceerd in:Muscle Nerve
Hoofdauteurs: Dyck, P. James B., Coelho, Teresa, Waddington Cruz, Marcia, Brannagan, Thomas H., Khella, Sami, Karam, Chafic, Berk, John L., Polydefkis, Michael J., Kincaid, John C., Wiesman, Janice F., Litchy, William J., Mauermann, Michelle L., Ackermann, Elizabeth J., Baker, Brenda F., Jung, Shiangtung W., Guthrie, Spencer, Pollock, Michael, Dyck, Peter J.
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: John Wiley & Sons, Inc. 2020
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Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7540369/
https://ncbi.nlm.nih.gov/pubmed/32654156
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/mus.27023
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