TTR kinetic stabilizers and TTR gene silencing: A new era in therapy for familial amyloidotic polyneuropathies

Expert Opinion on Pharmacotherapy

Volumn 17, Issue 6, 2016, Pages 791-802

  Adams, David ^a,b,c   Cauquil, Cecile ^a,b,c   Labeyrie, Céline ^a,b,c   Beaudonnet, Guillemette ^a,b,c   Algalarrondo, Vincent ^b,c,d   Théaudin, Marie ^a,b,c  

^a BICÊTRE HOSPITAL   (France)

^b National Reference Center for FAP (NNERF)   (France)

^c FILNEMUS Filière Nationale des Maladies Neuromusculaires   (France)

^d HÔPITAL ANTOINE BÉCLÈRE   (France)

Author keywords

ALN TTR02 patisiran; diflunisal; familial amyloidotic polyneuropathy; ISIS TTR Rx; liver transplantation; Mab GSK2398852; tafamidis; Transthyretin; TTR gene silencing; TTR kinetic stabilizer

Indexed keywords

AMYLOID; ANTISENSE OLIGONUCLEOTIDE; CREATININE; DIFLUNISAL; MONOCLONAL ANTIBODY; PREALBUMIN; SERUM AMYLOID P; SMALL INTERFERING RNA; TAFAMIDIS; ALBUMIN RECEPTOR; BENZOXAZOLE DERIVATIVE; TRANSTHYRETIN RECEPTOR;

CLINICAL ASSESSMENT TOOL; CREATININE CLEARANCE; DIARRHEA; DISEASE COURSE; DISEASE SEVERITY; DRUG EFFICACY; DRUG MARKETING; DRUG SAFETY; DRUG TOLERABILITY; FAMILIAL AMYLOID POLYNEUROPATHY; FECES INCONTINENCE; GENE SILENCING; HUMAN; KIDNEY DYSFUNCTION; LIVER TRANSPLANTATION; MEDLINE; MULTICENTER STUDY (TOPIC); NONHUMAN; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); PHASE 3 CLINICAL TRIAL (TOPIC); PHENOTYPE; RANDOMIZED CONTROLLED TRIAL (TOPIC); REVIEW; RNAI THERAPEUTICS; THROMBOCYTOPENIA; TREATMENT INDICATION; URINARY TRACT INFECTION; AMYLOID NEUROPATHIES, FAMILIAL; GENETICS; METABOLISM;

AMYLOID NEUROPATHIES, FAMILIAL; BENZOXAZOLES; CLINICAL TRIALS, PHASE III AS TOPIC; DIFLUNISAL; DISEASE PROGRESSION; GENE SILENCING; HUMANS; LIVER TRANSPLANTATION; PREALBUMIN; RECEPTORS, ALBUMIN;

EID: 84961203990     PISSN: 14656566     EISSN: 17447666     Source Type: Journal    
DOI: 10.1517/14656566.2016.1145664     Document Type: Review

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