Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose study

Orphanet Journal of Rare Diseases

Volumn 10, Issue 1, 2015, Pages

  Suhr, Ole B ^a   Coelho, Teresa ^b   Buades, Juan ^c   Pouget, Jean ^d   Conceicao, Isabel ^e   Berk, John ^f   Schmidt, Hartmut ^g   Waddington Cruz, Márcia ^h   Campistol, Josep M ⁱ   Bettencourt, Brian R ^j   Vaishnaw, Akshay ^j   Gollob, Jared ^j   Adams, David ^k  

^a UMEÅ UNIVERSITY   (Sweden)

^b HOSPITAL DE SANTO ANTÓNIO   (Portugal)

^c HOSPITAL SON LLÀTZER   (Spain)

^d TIMONE HOSPITAL   (France)

^e HOSPITAL DE SANTA MARIA   (Portugal)

^f BOSTON UNIVERSITY   (United States)

^g UNIVERSITY HOSPITAL MÜNSTER   (Germany)

^h FEDERAL UNIVERSITY OF RIO DE JANEIRO   (Brazil)

ⁱ UNIVERSITY OF BARCELONA   (Spain)

^j ALNYLAM PHARMACEUTICALS   (United States)

^k BICÊTRE HOSPITAL   (France)

more..

Author keywords

Clinical trial; Genetic mutation; Hereditary disease; Patisiran; Phase II; Polyneuropathy; RNA interference; Transthyretin mediated familial amyloidotic polyneuropathy

Indexed keywords

ALPHA INTERFERON; GAMMA INTERFERON INDUCIBLE PROTEIN 10; GRANULOCYTE COLONY STIMULATING FACTOR; INTERLEUKIN 1 RECEPTOR BLOCKING AGENT; INTERLEUKIN 12; INTERLEUKIN 1BETA; INTERLEUKIN 6; PATISIRAN; TUMOR NECROSIS FACTOR ALPHA; SMALL INTERFERING RNA;

ABDOMINAL PAIN; ADULT; AGED; AREA UNDER THE CURVE; ARTICLE; ASTHENIA; BACKACHE; BRONCHOSPASM; CELLULITIS; CHILL; CLINICAL ARTICLE; CONTROLLED STUDY; DISEASE SEVERITY; DIZZINESS; DOSE RESPONSE; DRUG DOSE ESCALATION; DRUG EFFICACY; DRUG HALF LIFE; DRUG SAFETY; DRUG TOLERABILITY; DYSPHAGIA; DYSPNEA; ERYTHEMA; FACE ERYTHEMA; FAMILIAL AMYLOID POLYNEUROPATHY; FEMALE; FEVER; HUMAN; INFUSION RELATED REACTION; INTENTION TO TREAT ANALYSIS; LEUKOCYTOSIS; LYMPHANGITIS; MALE; MAXIMUM PLASMA CONCENTRATION; MULTICENTER STUDY; NAUSEA; NEUTROPHILIA; OXYGEN SATURATION; PALLOR; PHASE 2 CLINICAL TRIAL; POLYURIA; PROTEIN BLOOD LEVEL; SEPSIS; TACHYCARDIA; TACHYPNEA; TREATMENT WITHDRAWAL; UNSPECIFIED SIDE EFFECT; URINARY TRACT INFECTION; VOMITING; XEROSTOMIA; AMYLOID NEUROPATHIES, FAMILIAL; CLINICAL TRIAL; MIDDLE AGED;

AGED; AMYLOID NEUROPATHIES, FAMILIAL; DOSE-RESPONSE RELATIONSHIP, DRUG; FEMALE; HUMANS; MALE; MIDDLE AGED; RNA, SMALL INTERFERING;

EID: 84940759062     PISSN: None     EISSN: 17501172     Source Type: Journal    
DOI: 10.1186/s13023-015-0326-6     Document Type: Article

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