References
- AndradeCA peculiar form of peripheral neuropathy; familiar atypical generalized amyloidosis with special involvement of the peripheral nervesBrain195275340842712978172
- WestermarkPSlettenKJohanssonBCornwellGGFibril in senile systemic amyloidosis is derived from normal transthyretinProc Natl Acad Sci U S A1990877284328452320592
- WangAKGorevicPDNeurologic improvement after cervical decompression in wild type transthyretin amyloidosisDyckPJCompanion to Peripheral NeurologyPhiladelphia, PA, USASaunders2010145147
- BensonMDKincaidJCThe molecular biology and clinical features of amyloid neuropathyMuscle Nerve200736441142317554795
- GertzMABensonMDDyckPJDiagnosis, prognosis, and therapy of transthyretin amyloidosisJ Am Coll Cardiol201566212451246626610878
- Plante-BordeneuveVTransthyretin familial amyloid polyneuropathy: an updateJ Neurol2018265497698329249054
- SchmidtHHWaddington-CruzMBottemanMFEstimating the global prevalence of transthyretin familial amyloid polyneuropathyMuscle Nerve201857582983729211930
- HawkinsPNAndoYDispenzeriAGonzalez-DuarteAAdamsDSuhrOBEvolving landscape in the management of transthyretin amyloidosisAnn Med201547862563826611723
- WallaceMRNaylorSLKluve-BeckermanBLocalization of the human prealbumin gene to chromosome 18Biochem Biophys Res Commun198512937537582990465
- BuxbaumJNOligonucleotide drugs for transthyretin amyloidosisN Engl J Med20183791828529972750
- NiemietzCChandhokGSchmidtHTherapeutic oligonucleotides targeting liver disease: TTR amyloidosisMolecules20152010179441797526437390
- MerliniGBellottiVMolecular mechanisms of amyloidosisN Engl J Med2003349658359612904524
- HolmgrenGSteenLSuhrOClinical improvement and amyloid regression after liver transplantation in hereditary transthyretin amyloidosisLancet19933418853111311168097803
- BensonMDLiver transplantation and transthyretin amyloidosisMuscle Nerve201347215716223169427
- Familial Amyloid Polyneuropathy World Transplant Registry Available from: http://fapwtr.orgAccessed September 11, 2018
- EriczonBGSuhrOBrooméULiver transplantation halts the progress of familial amyloidotic polyneuropathyTransplant Proc199527112337878861
- LiepnieksJJZhangLQBensonMDProgression of transthyretin amyloid neuropathy after liver transplantationNeurology201075432432720660862
- YazakiMTokudaTNakamuraACardiac amyloid in patients with familial amyloid polyneuropathy consists of abundant wild-type transthyretinBiochem Biophys Res Commun2000274370270610924339
- YazakiMLiepnieksJJKincaidJCBensonMDContribution of wild-type transthyretin to hereditary peripheral nerve amyloidMuscle Nerve200328443844214506715
- JohnsonSMConnellySFearnsCPowersETKellyJWThe transthyretin amyloidoses: from delineating the molecular mechanism of aggregation linked to pathology to a regulatory-agency-approved drugJ Mol Biol20124212–318520322244854
- SekijimaYDendleMAKellyJWOrally administered diflunisal stabilizes transthyretin against dissociation required for amyloidogenesisAmyloid200613423624917107884
- BerkJLSuhrOBObiciLRepurposing diflunisal for familial amyloid polyneuropathy: a randomized clinical trialJAMA2013310242658266724368466
- CoelhoTMaiaLFMartins da SilvaATafamidis for transthyretin familial amyloid polyneuropathy: a randomized, controlled trialNeurology201279878579222843282
- MaurerMSSchwartzJHGundapaneniBATTR-ACT Study InvestigatorsTafamidis treatment for patients with transthyretin amyloid cardiomyopathyN Engl J Med2018379111007101630145929
- PenchalaSCConnellySWangYAG10 inhibits amyloidogenesis and cellular toxicity of the familial amyloid cardiomyopathy-associated V122I transthyretinProc Natl Acad Sci U S A2013110249992999723716704
- ObiciLCorteseALozzaADoxycycline plus tauroursodeoxycholic acid for transthyretin amyloidosis: a phase II studyAmyloid201219Suppl 1343622551192
- PalhaJAHaysMTMorreale de EscobarGEpiskopouVGottesmanMESaraivaMJTransthyretin is not essential for thyroxine to reach the brain and other tissues in transthyretin-null miceAm J Physiol19972723 Pt 1E485E4939124556
- CrookeSTWitztumJLBennettCFBakerBFRNA-targeted therapeuticsCell Metab201827471473929617640
- ShenXCoreyDRChemistry, mechanism and clinical status of anti-sense oligonucleotides and duplex RNAsNucleic Acids Res20184641584160029240946
- BennettCFBakerBFPhamNSwayzeEGearyRSPharmacology of antisense drugsAnnu Rev Pharmacol Toxicol20175718110527732800
- GoyalNNarayanaswamiPMaking sense of antisense oligonucleotides: a narrative reviewMuscle Nerve201857335637029105153
- AdamsDGonzalez-DuarteAO’RiordanWDPatisiran, an RNAi therapeutic, for hereditary transthyretin amyloidosisN Engl J Med20183791112129972753
- BensonMDWaddington-CruzMBerkJLInotersen treatment for patients with hereditary transthyretin amyloidosisN Engl J Med20183791223129972757
- RossorAMReillyMMSleighJNAntisense oligonucleotides and other genetic therapies made simplePract Neurol201818212613129455156
- CrookeSTMolecular mechanisms of antisense oligonucleotidesNucleic Acid Ther2017272707728080221
- SuhrOBCoelhoTBuadesJEfficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose studyOrphanet J Rare Dis20151011925603901
- FDA approves first-of-its kind targeted RNA-based therapy to treat a rare diseaseFDA news release August 10 20182018 Available from: https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm616518.htmAccessed September 11, 2018
- BensonMDPandeySWitchellDAntisense oligonucleotide therapy for TTR amyloidosisAmyloid201118Suppl 16021838433
- AckermannEJGuoSBootenSClinical development of an antisense therapy for the treatment of transthyretin-associated poly-neuropathyAmyloid201219Suppl 1434422494066
- AckermannEJGuoSBensonMDSuppressing transthyretin production in mice, monkeys and humans using 2nd-generation anti-sense oligonucleotidesAmyloid201623314815727355239
- BrannaganTWangAKCoehloTOpen-label Extension of the Phase 3 NEURO-TTR Study to assess the long-term efficacy and safety of Inotersen in patients with hereditary transthyretin amyloidosisNeurology20189015 Supplement1.234
- KeamSJInotersen: first global approvalDrugs201878131371137630120737
- Press Release Available from: https://ir.akceatx.com/news-releases/news-release-details/akcea-and-ionis-receive-fda-approval-tegseditm-inotersenAccessed September 11, 2018
- BensonMDDasguptaNRRissingSMSmithJFeigenbaumHSafety and efficacy of a TTR specific antisense oligonucleotide in patients with transthyretin amyloid cardiomyopathyAmyloid201724421722328868918
- SchmidtHHBarrosoFGonzález-DuarteAManagement of asymptomatic gene carriers of transthyretin familial amyloid polyneuropathyMuscle Nerve201654335336027273296
- HabkaDMannDLandesRSoto-GutierrezAFuture economics of liver transplantation: a 20-year cost modeling forecast and the prospect of bioengineering autologous liver graftsPLoS One2015107e01317642126177505
- StewartMShafferSMurphyBCharacterizing the high disease burden of transthyretin amyloidosis for patients and caregiversNeurol Ther20187234936430073497