Fortanier E, Michel C, Hostin M, Delmont E, Verschueren A, Guye M
Eur J Neurol. 2024; 32(1):e16574.
PMID: 39601438
PMC: 11625930.
DOI: 10.1111/ene.16574.
Karimi M, Esmaeilpour Moallem F, Gholami Chahkand M, Azarm E, Emami Kazemabad M, Dadkhah P
Front Neurol. 2024; 15:1465747.
PMID: 39286810
PMC: 11402727.
DOI: 10.3389/fneur.2024.1465747.
Gentile L, Diemberger I, Plante-Bordeneuve V, Mazzeo A, Dori A, Luigetti M
PLoS One. 2024; 19(1):e0292435.
PMID: 38241252
PMC: 10798432.
DOI: 10.1371/journal.pone.0292435.
Gonzalez-Moreno J, Dispenzieri A, Grogan M, Coelho T, Tournev I, Waddington-Cruz M
Cardiol Ther. 2023; 13(1):117-135.
PMID: 38117424
PMC: 10899146.
DOI: 10.1007/s40119-023-00344-3.
Gentile L, Coelho T, Dispenzieri A, Conceicao I, Waddington-Cruz M, Kristen A
Orphanet J Rare Dis. 2023; 18(1):350.
PMID: 37946256
PMC: 10636983.
DOI: 10.1186/s13023-023-02962-5.
Optimal practices for the management of hereditary transthyretin amyloidosis: real-world experience from Japan, Brazil, and Portugal.
Ando Y, Waddington-Cruz M, Sekijima Y, Koike H, Ueda M, Konishi H
Orphanet J Rare Dis. 2023; 18(1):323.
PMID: 37828588
PMC: 10571420.
DOI: 10.1186/s13023-023-02910-3.
Impact of Vutrisiran on Quality of Life and Physical Function in Patients with Hereditary Transthyretin-Mediated Amyloidosis with Polyneuropathy.
Obici L, Ajroud-Driss S, Lin K, Berk J, Gillmore J, Kale P
Neurol Ther. 2023; 12(5):1759-1775.
PMID: 37523143
PMC: 10444729.
DOI: 10.1007/s40120-023-00522-4.
Amylo-AFFECT-QOL, a self-reported questionnaire to assess health-related quality of life and to determine the prognosis in cardiac amyloidosis.
Kharoubi M, Bezard M, Broussier A, Galat A, Gounot R, Poullot E
Front Cardiovasc Med. 2023; 10:1124660.
PMID: 36998975
PMC: 10043221.
DOI: 10.3389/fcvm.2023.1124660.
Inotersen to Treat Polyneuropathy Associated with Hereditary Transthyretin (hATTR) Amyloidosis.
Robinson C, Pham C, Zamarripa A, Dugay C, Lee C, Berger A
Health Psychol Res. 2023; 10(5):67910.
PMID: 36726478
PMC: 9886172.
DOI: 10.52965/001c.67910.
Long-term efficacy and safety of inotersen for hereditary transthyretin amyloidosis: NEURO-TTR open-label extension 3-year update.
Brannagan T, Coelho T, Wang A, Polydefkis M, Dyck P, Berk J
J Neurol. 2022; 269(12):6416-6427.
PMID: 35908242
PMC: 9618524.
DOI: 10.1007/s00415-022-11276-8.
Clinical and genetic profile of patients enrolled in the Transthyretin Amyloidosis Outcomes Survey (THAOS): 14-year update.
Dispenzieri A, Coelho T, Conceicao I, Waddington-Cruz M, Wixner J, Kristen A
Orphanet J Rare Dis. 2022; 17(1):236.
PMID: 35717381
PMC: 9206752.
DOI: 10.1186/s13023-022-02359-w.
Gly83Arg Mutation: Beyond Familial Vitreous Amyloidosis.
Li Z, Du K, Chu X, Lv H, Zhang W, Wang Z
Front Neurol. 2022; 12:821003.
PMID: 35185758
PMC: 8850374.
DOI: 10.3389/fneur.2021.821003.
Early Data on Long-term Impact of Inotersen on Quality-of-Life in Patients with Hereditary Transthyretin Amyloidosis Polyneuropathy: Open-Label Extension of NEURO-TTR.
Yarlas A, Lovley A, McCausland K, Brown D, Vera-Llonch M, Conceicao I
Neurol Ther. 2021; 10(2):865-886.
PMID: 34355354
PMC: 8571454.
DOI: 10.1007/s40120-021-00268-x.
Phenotypic Differences of Glu89Gln Genotype in ATTR Amyloidosis From Endemic Loci: Update From THAOS.
Gentile L, Tournev I, Amass L, Chapman D, Mazzeo A
Cardiol Ther. 2021; 10(2):481-490.
PMID: 34148211
PMC: 8555027.
DOI: 10.1007/s40119-021-00226-6.
Responder analysis for neuropathic impairment and quality-of-life assessment in patients with hereditary transthyretin amyloidosis with polyneuropathy in the NEURO-TTR study.
Yarlas A, Lovley A, Brown D, Kosinski M, Vera-Llonch M
J Neurol. 2021; 269(1):323-335.
PMID: 34125267
PMC: 8739287.
DOI: 10.1007/s00415-021-10635-1.
ATTR amyloidosis during the COVID-19 pandemic: insights from a global medical roundtable.
Brannagan 3rd T, Auer-Grumbach M, Berk J, Briani C, Bril V, Coelho T
Orphanet J Rare Dis. 2021; 16(1):204.
PMID: 33957949
PMC: 8100737.
DOI: 10.1186/s13023-021-01834-0.
Screening for ATTR amyloidosis in the clinic: overlapping disorders, misdiagnosis, and multiorgan awareness.
Nativi-Nicolau J, Karam C, Khella S, Maurer M
Heart Fail Rev. 2021; 27(3):785-793.
PMID: 33609196
PMC: 9033715.
DOI: 10.1007/s10741-021-10080-2.
Patient and family experience with transthyretin amyloid cardiomyopathy (ATTR-CM) and polyneuropathy (ATTR-PN) amyloidosis: results of two focus groups.
Rintell D, Heath D, Braga Mendendez F, Cross E, Cross T, Knobel V
Orphanet J Rare Dis. 2021; 16(1):70.
PMID: 33557882
PMC: 7869246.
DOI: 10.1186/s13023-021-01706-7.
Early data on long-term efficacy and safety of inotersen in patients with hereditary transthyretin amyloidosis: a 2-year update from the open-label extension of the NEURO-TTR trial.
Brannagan T, Wang A, Coelho T, Waddington Cruz M, Polydefkis M, Dyck P
Eur J Neurol. 2020; 27(8):1374-1381.
PMID: 32343462
PMC: 7496583.
DOI: 10.1111/ene.14285.
Pharmacological treatment for familial amyloid polyneuropathy.
Magrinelli F, Fabrizi G, Santoro L, Manganelli F, Zanette G, Cavallaro T
Cochrane Database Syst Rev. 2020; 4:CD012395.
PMID: 32311072
PMC: 7170468.
DOI: 10.1002/14651858.CD012395.pub2.
