Research & Literature

Free light chain testing for the diagnosis, monitoring and prognostication of AL amyloidosis.

Mollee P, Merlini G

Clinical chemistry and laboratory medicine 2016; (54(6)):921-7.

[Presentations of transthyretin associated familial amyloid polyneuropathy in Argentina].

Chaves M, Bettini M, Marciano S, et al.

Medicina 2016; (76(2)):105-8.

Schwann cell and endothelial cell damage in transthyretin familial amyloid polyneuropathy.

Koike H, Ikeda S, Takahashi M, et al.

Neurology 2016; (87(21)):2220-2229 doi:10.1212/WNL.0000000000003362.

Current and Future Treatment Approaches in Transthyretin Familial Amyloid Polyneuropathy.

Kerschen P, Planté-Bordeneuve V

Current treatment options in neurology 2016; (18(12)):53 doi:10.1007/s11940-016-0436-z.

Elderly onset vitreous opacities as the initial manifestation in hereditary transthyretin (ATTR Val30Met) carries.

Ishida K, Nishida T, Niimi Y, et al.

Ophthalmic genetics 2017; (38(4)):387-391 doi:10.1080/13816810.2016.1232413.

Treatment of transthyretin familial amyloid polyneuropathy with tafamidis: a case report.

Miyazaki Y

Nihon Ronen Igakkai zasshi. Japanese journal of geriatrics 2017; (54(1)):75-80 doi:10.3143/geriatrics.54.75.

Nucleobindin 1 binds to multiple types of pre-fibrillar amyloid and inhibits fibrillization.

Bonito-Oliva A, Barbash S, Sakmar TP, Graham WV

Scientific reports 2017; (7()):42880 doi:10.1038/srep42880.

Impact of genotype and phenotype on cardiac biomarkers in patients with transthyretin amyloidosis - Report from the Transthyretin Amyloidosis Outcome Survey (THAOS).

Kristen AV, Maurer MS, Rapezzi C, et al.

PloS one 2017; (12(4)):e0173086 doi:10.1371/journal.pone.0173086.

Progression of myocardial sympathetic denervation assessed by 123I-MIBG imaging in familial amyloid polyneuropathy and the effect of liver transplantation.

Azevedo Coutinho MDC, Cortez-Dias N, Cantinho G, et al.

Revista portuguesa de cardiologia : orgao oficial da Sociedade Portuguesa de Cardiologia = Portuguese journal of cardiology : an official journal of the Portuguese Society of Cardiology 2017; (36(5)):333-340 doi:10.1016/j.repc.2016.08.010.

Design and Rationale of the Phase 3 ATTR-ACT Clinical Trial (Tafamidis in Transthyretin Cardiomyopathy Clinical Trial).

Maurer MS, Elliott P, Merlini G, et al.

Circulation. Heart failure 2017; (10(6)) doi:10.1161/CIRCHEARTFAILURE.116.003815.

Semi-quantitative models for identifying potent and selective transthyretin amyloidogenesis inhibitors.

Connelly S, Mortenson DE, Choi S, et al.

Bioorganic & medicinal chemistry letters 2017; (27(15)):3441-3449 doi:10.1016/j.bmcl.2017.05.080.

Subjects at Risk for Genetic Late-Onset Neurological Diseases: Objective Knowledge.

Leite Â, Leite F, Dinis MAP

Public health genomics 2017; (20(3)):158-165 doi:10.1159/000479292.

Cardiac and peripheral vasomotor autonomic functions in late-onset transthyretin Val30Met familial amyloid polyneuropathy.

Koike H, Nakamura T, Hashizume A, et al.

Journal of neurology 2017; (264(11)):2293-2302 doi:10.1007/s00415-017-8629-2.

Frequency of and Prognostic Significance of Cardiac Involvement at Presentation in Hereditary Transthyretin-Derived Amyloidosis and the Value of N-Terminal Pro-B-Type Natriuretic Peptide.

Klaassen SHC, Tromp J, Nienhuis HLA, et al.

The American journal of cardiology 2018; (121(1)):107-112 doi:10.1016/j.amjcard.2017.09.029.

Diagnosis and management of transthyretin familial amyloid polyneuropathy in Japan: red-flag symptom clusters and treatment algorithm.

Sekijima Y, Ueda M, Koike H, et al.

Orphanet journal of rare diseases 2018; (13(1)):6 doi:10.1186/s13023-017-0726-x.

Autonomic involvement in hereditary transthyretin amyloidosis (hATTR amyloidosis).

Gonzalez-Duarte A

Clinical autonomic research : official journal of the Clinical Autonomic Research Society 2019; (29(2)):245-251 doi:10.1007/s10286-018-0514-2.

The genetic heterogeneity of hereditary transthyretin amyloidosis in a sample of the Brazilian population.

Lavigne-Moreira C, Marques VD, Gonçalves MVM, et al.

Journal of the peripheral nervous system : JPNS 2018; (23(2)):134-137 doi:10.1111/jns.12259.

Single-centre experience on transthyretin familial amyloid polyneuropathy: case series and literature review.

Martens B, De Pauw M, De Bleecker JL

Acta neurologica Belgica 2018; (118(2)):179-185 doi:10.1007/s13760-018-0906-z.

[Transthyretin Familial Amyloid Polyneuropathy - Disease Profile of a Multisystem Disorder].

Niemietz C, Röcken C, Schilling M, et al.

Deutsche medizinische Wochenschrift (1946) 2018; (143(6)):427-430 doi:10.1055/s-0043-123681.

Long-term predictors for psychological outcome of pre-symptomatic testing for late-onset neurological diseases.

Lêdo S, Ramires A, Leite Â, et al.

European journal of medical genetics 2018; (61(10)):575-580 doi:10.1016/j.ejmg.2018.03.010.

Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis.

Benson MD, Waddington-Cruz M, Berk JL, et al.

The New England journal of medicine 2018; (379(1)):22-31 doi:10.1056/NEJMoa1716793.

New Medications in the Treatment of Hereditary Transthyretin Amyloidosis.

Walker S

Hospital pharmacy 2018; (53(4)):236-238 doi:10.1177/0018578718779757.

Kind and distribution of cutaneous sensation loss in hereditary transthyretin amyloidosis with polyneuropathy.

Pinto MV, Dyck PJB, Gove LE, et al.

Journal of the neurological sciences 2018; (394()):78-83 doi:10.1016/j.jns.2018.08.031.

Hereditary transthyretin-related amyloidosis.

Finsterer J, Iglseder S, Wanschitz J, et al.

Acta neurologica Scandinavica 2019; (139(2)):92-105 doi:10.1111/ane.13035.

Late-onset Transthyretin (TTR)-familial Amyloid Polyneuropathy (FAP) with a Long Disease Duration from Non-endemic Areas in Japan.

Miyake Z, Nakamagoe K, Ezawa N, et al.

Internal medicine (Tokyo, Japan) 2019; (58(5)):713-718 doi:10.2169/internalmedicine.1457-18.

Origin of sporadic late-onset hereditary ATTR Val30Met amyloidosis in Japan.

Ueda M, Yamashita T, Misumi Y, et al.

Amyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis 2018; (25(3)):143-147 doi:10.1080/13506129.2018.1531842.

An indirect treatment comparison of the efficacy of patisiran and tafamidis for the treatment of hereditary transthyretin-mediated amyloidosis with polyneuropathy.

Planté-Bordeneuve V, Lin H, Gollob J, et al.

Expert opinion on pharmacotherapy 2019; (20(4)):473-481 doi:10.1080/14656566.2018.1554648.

Hereditary cardiac amyloidosis associated with Pro24Ser transthyretin mutation: a case report.

Yamamoto H, Hashimoto T, Kawamura S, et al.

Journal of medical case reports 2018; (12(1)):370 doi:10.1186/s13256-018-1931-5.

Ultrastructure in Transthyretin Amyloidosis: From Pathophysiology to Therapeutic Insights.

Koike H, Katsuno M

Biomedicines 2019; (7(1)) doi:10.3390/biomedicines7010011.

Baseline disease characteristics in Brazilian patients enrolled in Transthyretin Amyloidosis Outcome Survey (THAOS).

Cruz MW, Pinto MV, Pinto LF, et al.

Arquivos de neuro-psiquiatria 2019; (77(2)):96-100 doi:10.1590/0004-282X20180156.

New insights into the clinical evaluation of hereditary transthyretin amyloidosis patients: a single center's experience.

Suhr OB, Gustavsson S, Heldestad V, et al.

Degenerative neurological and neuromuscular disease 2012; (2()):93-106 doi:10.2147/DNND.S24652.

Twenty Years of a Pre-Symptomatic Testing Protocol for Late-Onset Neurological Diseases in Portugal.

Paneque M, Félix J, Mendes Á, et al.

Acta medica portuguesa 2019; (32(4)):295-304 doi:10.20344/amp.10526.

Tegsedi (Inotersen): An Antisense Oligonucleotide Approved for the Treatment of Adult Patients with Hereditary Transthyretin Amyloidosis.

Gales L

Pharmaceuticals (Basel, Switzerland) 2019; (12(2)) doi:10.3390/ph12020078.

Inotersen: new promise for the treatment of hereditary transthyretin amyloidosis.

Mathew V, Wang AK

Drug design, development and therapy 2019; (13()):1515-1525 doi:10.2147/DDDT.S162913.

Inotersen for the treatment of adults with polyneuropathy caused by hereditary transthyretin-mediated amyloidosis.

Gertz MA, Scheinberg M, Waddington-Cruz M, et al.

Expert review of clinical pharmacology 2019; (12(8)):701-711 doi:10.1080/17512433.2019.1635008.

Patisiran Pharmacokinetics, Pharmacodynamics, and Exposure-Response Analyses in the Phase 3 APOLLO Trial in Patients With Hereditary Transthyretin-Mediated (hATTR) Amyloidosis.

Zhang X, Goel V, Attarwala H, et al.

Journal of clinical pharmacology 2020; (60(1)):37-49 doi:10.1002/jcph.1480.

Advances in the treatment of hereditary transthyretin amyloidosis: A review.

Gertz MA, Mauermann ML, Grogan M, Coelho T

Brain and behavior 2019; (9(9)):e01371 doi:10.1002/brb3.1371.

Development of measures of polyneuropathy impairment in hATTR amyloidosis: From NIS to mNIS + 7.

Dyck PJB, González-Duarte A, Obici L, et al.

Journal of the neurological sciences 2019; (405()):116424 doi:10.1016/j.jns.2019.116424.

Orthostatic hypotension in hereditary transthyretin amyloidosis: epidemiology, diagnosis and management.

Palma JA, Gonzalez-Duarte A, Kaufmann H

Clinical autonomic research : official journal of the Clinical Autonomic Research Society 2019; (29(Suppl 1)):33-44 doi:10.1007/s10286-019-00623-x.

Diagnosis and treatment of gastrointestinal dysfunction in hereditary TTR amyloidosis.

Obici L, Suhr OB

Clinical autonomic research : official journal of the Clinical Autonomic Research Society 2019; (29(Suppl 1)):55-63 doi:10.1007/s10286-019-00628-6.

Phenotypes of Late-Onset Transthyretin Amyloid Neuropathy: A Diagnostic Challenge.

Živković SA, Mnatsakanova D, Lacomis D

Journal of clinical neuromuscular disease 2019; (21(1)):1-6 doi:10.1097/CND.0000000000000252.

Strategies to improve the quality of life in patients with hereditary transthyretin amyloidosis (hATTR) and autonomic neuropathy.

Gendre T, Planté-Bordeneuve V

Clinical autonomic research : official journal of the Clinical Autonomic Research Society 2019; (29(Suppl 1)):25-31 doi:10.1007/s10286-019-00624-w.

Carpal tunnel syndrome and associated symptoms as first manifestation of hATTR amyloidosis.

Karam C, Dimitrova D, Christ M, Heitner SB

Neurology. Clinical practice 2019; (9(4)):309-313 doi:10.1212/CPJ.0000000000000640.

Neuropathy Associated with Systemic Amyloidosis.

Kaku M, Berk JL

Seminars in neurology 2019; (39(5)):578-588 doi:10.1055/s-0039-1688994.

Amyloidosis in Heart Failure.

Ihne S, Morbach C, Obici L, et al.

Current heart failure reports 2019; (16(6)):285-303 doi:10.1007/s11897-019-00446-x.

Gastrointestinal Manifestations in Hereditary Transthyretin Amyloidosis associated with Glu89Gln Mutation.

Nakov R, Sarafov S, Nakov V, et al.

Journal of gastrointestinal and liver diseases : JGLD 2019; (28(4)):421-426 doi:10.15403/jgld-362.

Hereditary transthyretin amyloidosis caused by the rare Phe33Leu mutation.

Björkenheim A, Szabó B, Sztaniszláv ÁJ

BMJ case reports 2020; (13(1)) doi:10.1136/bcr-2019-232756.

Diagnosis and Treatment of Hereditary Transthyretin Amyloidosis (hATTR) Polyneuropathy: Current Perspectives on Improving Patient Care.

Luigetti M, Romano A, Di Paolantonio A, et al.

Therapeutics and clinical risk management 2020; (16()):109-123 doi:10.2147/TCRM.S219979.

Population Pharmacokinetic-Pharmacodynamic Modeling of Inotersen, an Antisense Oligonucleotide for Treatment of Patients with Hereditary Transthyretin Amyloidosis.

Yu RZ, Collins JW, Hall S, et al.

Nucleic acid therapeutics 2020; (30(3)):153-163 doi:10.1089/nat.2019.0822.

The sensitivity of DPD scintigraphy to detect transthyretin cardiac amyloidosis in V30M mutation depends on the phenotypic expression of the disease.

Azevedo Coutinho MC, Cortez-Dias N, Cantinho G, et al.

Amyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis 2020; (27(3)):174-183 doi:10.1080/13506129.2020.1744553.

Hereditary transthyretin amyloidosis: current treatment.

Adams D, Slama M

Current opinion in neurology 2020; (33(5)):553-561 doi:10.1097/WCO.0000000000000852.

Neurofilament light chain, a biomarker for polyneuropathy in systemic amyloidosis.

Louwsma J, Brunger AF, Bijzet J, et al.

Amyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis 2021; (28(1)):50-55 doi:10.1080/13506129.2020.1815696.

Red flags in patients with hereditary transthyretin amyloidosis at diagnosis in a non-endemic area of Spain.

Silva-Hernández L, Horga Hernández A, Valls Carbó A, et al.

Neurologia 2020; doi:10.1016/j.nrl.2020.06.009.

Clinicopathological correlations of sural nerve biopsies in TTR Val30Met familial amyloid polyneuropathy.

Fernandes A, Coelho T, Rodrigues A, et al.

Brain communications 2019; (1(1)):fcz032 doi:10.1093/braincomms/fcz032.

hATTR Pathology: Nerve Biopsy Results from Italian Referral Centers.

Luigetti M, Romozzi M, Bisogni G, et al.

Brain sciences 2020; (10(11)) doi:10.3390/brainsci10110780.

Patient-reported burden of hereditary transthyretin amyloidosis on functioning and well-being.

Lovley A, Raymond K, Guthrie SD, et al.

Journal of patient-reported outcomes 2021; (5(1)):3 doi:10.1186/s41687-020-00273-y.

A Review of Novel Agents and Clinical Considerations in Patients With ATTR Cardiac Amyloidosis.

Benbrahim M, Norman K, Sanchorawala V, et al.

Journal of cardiovascular pharmacology 2021; (77(5)):544-548 doi:10.1097/FJC.0000000000001004.

Exertional chest pain is sometimes more than just coronary atherosclerosis.

Morgado GJ, Gomes AC, Cruz IR, et al.

Revista portuguesa de cardiologia 2021; (40(3)):245.e1-245.e5 doi:10.1016/j.repc.2021.02.002.

Recent advances in the diagnosis and management of amyloid cardiomyopathy.

Nijst P, Tang WW

Faculty reviews 2021; (10()):31 doi:10.12703/r/10-31.

The neuropathy in hereditary transthyretin amyloidosis: A narrative review.

Tozza S, Severi D, Spina E, et al.

Journal of the peripheral nervous system : JPNS 2021; (26(2)):155-159 doi:10.1111/jns.12451.

Characteristics of Patients with Late- vs. Early-Onset Val30Met Transthyretin Amyloidosis from the Transthyretin Amyloidosis Outcomes Survey (THAOS).

Waddington-Cruz M, Wixner J, Amass L, et al.

Neurology and therapy 2021; (10(2)):753-766 doi:10.1007/s40120-021-00258-z.

Best Practices in Specialized Amyloidosis Centers in the United States: A Survey of Cardiologists, Nurses, Patients, and Patient Advocates.

Nativi-Nicolau J, Sarswat N, Fajardo J, et al.

Clinical Medicine Insights. Cardiology 2021; (15()):11795468211015230 doi:10.1177/11795468211015230.

Association between spinal stenosis and wild-type ATTR amyloidosis.

Godara A, Riesenburger RI, Zhang DX, et al.

Amyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis 2021; (28(4)):226-233 doi:10.1080/13506129.2021.1950681.

OCULAR ANGIOGRAPHIC FEATURES IN JAPANESE PATIENTS WITH VAL30MET HEREDITARY TRANSTHYRETIN AMYLOIDOSIS.

Kakihara S, Hirano T, Kitahara J, et al.

Retina (Philadelphia, Pa.) 2022; (42(1)):210-215 doi:10.1097/IAE.0000000000003291.

A challenging road to diagnosing transthyretin cardiac amyloidosis and using technetium-99m pyrophosphate bone scintigraphy in nuclear cardiology - A case report.

Kavita A, Abdul Onny MA, Suppiah S, et al.

The Medical journal of Malaysia 2021; (76(5)):762-767.

Expert opinion on monitoring symptomatic hereditary transthyretin-mediated amyloidosis and assessment of disease progression.

Adams D, Algalarrondo V, Polydefkis M, et al.

Orphanet journal of rare diseases 2021; (16(1)):411 doi:10.1186/s13023-021-01960-9.

Clinical and apparative investigation of large and small nerve fiber impairment in mixed cohort of ATTR-amyloidosis: impact on patient management and new insights in wild-type.

Papagianni A, Ihne S, Zeller D, et al.

Amyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis 2022; (29(1)):14-22 doi:10.1080/13506129.2021.1976751.

Hereditary transthyretin-mediated amyloidosis with polyneuropathy: baseline anthropometric, demographic and disease characteristics of patients from a reference center.

Sequeira VCC, Penetra MA, Duarte L, et al.

Arquivos de neuro-psiquiatria 2022; (80(3)):262-269 doi:10.1590/0004-282X-ANP-2020-0590.

Light Chain (AL) Cardiac Amyloidosis: A Diagnostic Dilemma.

Abdelazeem B, Manasrah N, Yousaf A, et al.

Cureus 2021; (13(11)):e19278 doi:10.7759/cureus.19278.

Skin amyloid deposits and nerve fiber loss as markers of neuropathy onset and progression in hereditary transthyretin amyloidosis.

Leonardi L, Adam C, Beaudonnet G, et al.

European journal of neurology 2022; (29(5)):1477-1487 doi:10.1111/ene.15268.

Familial Amyloid Polyneuropathy Misdiagnosed as Systemic Sclerosis.

Pereira MA, Lobão MM, Mesquita A, et al.

European journal of case reports in internal medicine 2022; (9(1)):003118 doi:10.12890/2022_003118.

Transthyretin: Its function and amyloid formation.

Ueda M

Neurochemistry international 2022; (155()):105313 doi:10.1016/j.neuint.2022.105313.

Novel approaches to diagnosis and management of hereditary transthyretin amyloidosis.

Carroll A, Dyck PJ, de Carvalho M, et al.

Journal of neurology, neurosurgery, and psychiatry 2022; (93(6)):668-678 doi:10.1136/jnnp-2021-327909.

Real-life experience with inotersen in hereditary transthyretin amyloidosis with late-onset phenotype: Data from an early-access program in Italy.

Luigetti M, Antonini G, Di Paolantonio A, et al.

European journal of neurology 2022; (29(7)):2148-2155 doi:10.1111/ene.15325.

Inotersen and severe thrombocytopenia: 2 case reports and review.

Domínguez Senín L, Borrachero Garro C, Sánchez Gómez E, Santos-Rubio MD

International journal of clinical pharmacology and therapeutics 2022; (60(7)):311-316 doi:10.5414/CP204190.

[Practice guideline for the treatment of familial amyloid polyneuropathy].

Carretero M, Sáez MS, Posadas-Martínez ML, et al.

Medicina 2022; (82(2)):262-274.

Clinical and Genetic Evaluation of People with or at Risk of Hereditary ATTR Amyloidosis: An Expert Opinion and Consensus on Best Practice in Ireland and the UK.

Gillmore JD, Reilly MM, Coats CJ, et al.

Advances in therapy 2022; (39(6)):2292-2301 doi:10.1007/s12325-022-02139-9.

Cardiac amyloidosis-interdisciplinary approach to diagnosis and therapy.

Hänselmann A, Berliner D, Bauersachs J, Bavendiek U

Herz 2022; (47(4)):324-331 doi:10.1007/s00059-022-05122-w.

Late-Onset Hereditary Transthyretin Amyloidosis Val30Met in an Elderly Person in a Non-Endemic Area.

Wang S, Sun J, Lu Q, et al.

International medical case reports journal 2022; (15()):299-306 doi:10.2147/IMCRJ.S357236.

Factors associated with increased health-related quality-of-life benefits in hereditary transthyretin amyloidosis polyneuropathy patients treated with inotersen.

Karam C, Brown D, Yang M, et al.

Muscle & nerve 2022; (66(3)):319-328 doi:10.1002/mus.27668.

Vutrisiran: First Approval.

Keam SJ

Drugs 2022; (82(13)):1419-1425 doi:10.1007/s40265-022-01765-5.

Treating hereditary transthyretin amyloidosis: Present & future challenges.

Echaniz-Laguna A, Cauquil C, Labeyrie C, Adams D

Revue neurologique 2023; (179(1-2)):30-34 doi:10.1016/j.neurol.2022.07.006.

Prompt diagnosis of a wild-type transthyretin cardiac amyloidosis: Role of multimodality imaging.

Lim SS, Kuo L, Chang FP, et al.

Journal of the Chinese Medical Association : JCMA 2022; (85(11)):1101-1105 doi:10.1097/JCMA.0000000000000817.

The use of PYP scan for evaluation of ATTR cardiac amyloidosis at a tertiary medical centre.

Dower J, Dima D, Lalla M, et al.

The British journal of cardiology 2022; (29(2)):19 doi:10.5837/bjc.2022.019.

Drug and Gene Therapy for Treating Variant Transthyretin Amyloidosis (ATTRv) Neuropathy.

Dardiotis E, Kyriakides T

Current neuropharmacology 2023; (21(3)):471-481 doi:10.2174/1570159X21666221108094736.

Red flags in patients with hereditary transthyretin amyloidosis at diagnosis in a non-endemic area of Spain.

Silva-Hernández L, Horga Hernández A, Valls Carbó A, et al.

Neurologia 2023; (38(2)):87-92 doi:10.1016/j.nrleng.2020.06.015.

Complications and Mortality Rate of Cytoreductive Surgery with Hyperthermic Intraperitoneal Chemotherapy: Italian Peritoneal Surface Malignancies Oncoteam Results Analysis.

Carboni F, Valle M, Vaira M, et al.

Cancers 2022; (14(23)) doi:10.3390/cancers14235824.

A case of severe increase of liver enzymes in a ATTRv patient after one year of inotersen treatment.

Severi D, Palumbo G, Spina E, et al.

Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology 2023; (44(4)):1419-1422 doi:10.1007/s10072-022-06568-w.

Neuropathic pain experience in symptomatic and presymptomatic subjects carrying a transthyretin gene mutation.

Tozza S, Luigetti M, Antonini G, et al.

Frontiers in neurology 2023; (14()):1109782 doi:10.3389/fneur.2023.1109782.

Indirect treatment comparison (ITC) of the efficacy of vutrisiran and tafamidis for hereditary transthyretin-mediated amyloidosis with polyneuropathy.

Merkel M, Danese D, Chen C, et al.

Expert opinion on pharmacotherapy 2023; (24(10)):1205-1214 doi:10.1080/14656566.2023.2215925.

Diagnosis for Chinese patients with light chain amyloidosis: a scoping review.

Chen M, Liu J, Wang X, et al.

Annals of medicine 2023; (55(1)):2227425 doi:10.1080/07853890.2023.2227425.

Pharmacokinetics and Pharmacodynamics of Patisiran in Patients with hATTR Amyloidosis and with Polyneuropathy After Liver Transplantation.

Badri P, Habtemariam B, Melch M, et al.

Clinical pharmacokinetics 2023; (62(10)):1509-1522 doi:10.1007/s40262-023-01292-w.

Serum neurofilament light chain: a promising early diagnostic biomarker for hereditary transthyretin amyloidosis?

Romano A, Primiano G, Antonini G, et al.

European journal of neurology 2024; (31(1)):e16070 doi:10.1111/ene.16070.

Optimal practices for the management of hereditary transthyretin amyloidosis: real-world experience from Japan, Brazil, and Portugal.

Ando Y, Waddington-Cruz M, Sekijima Y, et al.

Orphanet journal of rare diseases 2023; (18(1)):323 doi:10.1186/s13023-023-02910-3.

Detailed clinical, physiological and pathological phenotyping can impact access to disease-modifying treatments in ATTR carriers.

Beauvais D, Labeyrie C, Cauquil C, et al.

Journal of neurology, neurosurgery, and psychiatry 2024; (95(6)):489-499 doi:10.1136/jnnp-2023-332180.

Correlation between a commercial electrophysiological test of sudomotor function and intraepidermal nerve fiber density in hereditary transthyretin amyloidosis.

Masuda T, Misumi Y, Nomura T, et al.

Muscle & nerve 2024; (69(1)):99-102 doi:10.1002/mus.28005.

High-Sensitivity Cardiac Troponin T to Exclude Cardiac Involvement in TTR Variant Carriers and ATTRv Amyloidosis Patients.

Tingen HSA, Berends M, Tubben A, et al.

Journal of clinical medicine 2024; (13(3)) doi:10.3390/jcm13030810.

Longitudinal analysis of serum neurofilament light chain levels as marker for neuronal damage in hereditary transthyretin amyloidosis.

Berends M, Brunger AF, Bijzet J, et al.

Amyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis 2024; (31(2)):132-141 doi:10.1080/13506129.2024.2327342.

Hereditary Transthyretin Amyloidosis and the Impact of Classic and New Treatments on Kidney Function: A Review.

Meléndrez-Balcázar E, Aranda-Vela K, Cervantes-Hernández A, López-Cureño S

American journal of kidney diseases : the official journal of the National Kidney Foundation 2024; (84(2)):224-231 doi:10.1053/j.ajkd.2024.01.527.

Serum neurofilament light chain levels correlate with small fiber related parameters in patients with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN).

Galosi E, Costanzo R, Forcina F, et al.

Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology 2024; (45(10)):5023-5032 doi:10.1007/s10072-024-07562-0.

Clinical and imaging characteristics of patients with cardiac amyloidosis- a single center observational study.

Ingebrigtsen A, Saeed S, Larsen TH, Reikvam H

Scandinavian journal of clinical and laboratory investigation 2024; (84(3)):193-201 doi:10.1080/00365513.2024.2346908.

Genotype-Phenotype Correlations in ATTR Amyloidosis: A Clinical Update.

Monda E, Cirillo C, Verrillo F, et al.

Heart failure clinics 2024; (20(3)):317-323 doi:10.1016/j.hfc.2024.03.006.

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