臨床試験番号 NCT01960348 研究名 "APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis" - ClinicalTrials.gov
“Benzoxazoles as transthyretin amyloid fibril inhibitors: synthesis, evaluation, and mechanism of action”. Angew. Chem. Int. Ed. Engl.42 (24): 2758–61. (June 2003). doi:10.1002/anie.200351179. PMID12820260.
“Orally administered diflunisal stabilizes transthyretin against dissociation required for amyloidogenesis”. Amyloid13 (4): 236–49. (December 2006). doi:10.1080/13506120600960882. PMID17107884.
“Diflunisal analogues stabilize the native state of transthyretin. Potent inhibition of amyloidogenesis”. J. Med. Chem.47 (2): 355–74. (January 2004). doi:10.1021/jm030347n. PMID14711308.
Vilaro M, Arsequell G, Valencia G, Ballesteros A, Barluenga J, Nieto J, Planas A, Almeida R, Saraiva MJ (2007). “Reengineering TTR amyloid inhibition properties of diflunisal”. XIth International Symposium on Amyloidosis. Boca Raton: CRC. pp. 205–207. doi:10.1201/9781420043358.ch69. ISBN978-1-4200-4281-8
“Synthesis and evaluation of inhibitors of transthyretin amyloid formation based on the non-steroidal anti-inflammatory drug, flufenamic acid”. Bioorg. Med. Chem.7 (7): 1339–47. (July 1999). doi:10.1016/S0968-0896(99)00066-8. PMID10465408.
“The pathway by which the tetrameric protein transthyretin dissociates”. Biochemistry44 (47): 15525–33. (November 2005). doi:10.1021/bi051608t. PMID16300401.
“A peculiar form of peripheral neuropathy; familiar atypical generalized amyloidosis with special involvement of the peripheral nerves”. Brain75 (3): 408–27. (September 1952). doi:10.1093/brain/75.3.408. PMID12978172.
“Variant-sequence transthyretin (isoleucine 122) in late-onset cardiac amyloidosis in black Americans”. N. Engl. J. Med.336 (7): 466–73. (February 1997). doi:10.1056/NEJM199702133360703. PMID9017939.
“Partial denaturation of transthyretin is sufficient for amyloid fibril formation in vitro”. Biochemistry31 (36): 8654–60. (September 1992). doi:10.1021/bi00151a036. PMID1390650.
“The acid-mediated denaturation pathway of transthyretin yields a conformational intermediate that can self-assemble into amyloid”. Biochemistry35 (20): 6470–82. (May 1996). doi:10.1021/bi952501g. PMID8639594.
“Prevention of transthyretin amyloid disease by changing protein misfolding energetics”. Science299 (5607): 713–6. (January 2003). doi:10.1126/science.1079589. PMID12560553.
“An engineered transthyretin monomer that is nonamyloidogenic, unless it is partially denatured”. Biochemistry40 (38): 11442–52. (September 2001). doi:10.1021/bi011194d. PMID11560492.
“Benzoxazoles as transthyretin amyloid fibril inhibitors: synthesis, evaluation, and mechanism of action”. Angew. Chem. Int. Ed. Engl.42 (24): 2758–61. (June 2003). doi:10.1002/anie.200351179. PMID12820260.
“Orally administered diflunisal stabilizes transthyretin against dissociation required for amyloidogenesis”. Amyloid13 (4): 236–49. (December 2006). doi:10.1080/13506120600960882. PMID17107884.
“Diflunisal analogues stabilize the native state of transthyretin. Potent inhibition of amyloidogenesis”. J. Med. Chem.47 (2): 355–74. (January 2004). doi:10.1021/jm030347n. PMID14711308.
“Synthesis and evaluation of inhibitors of transthyretin amyloid formation based on the non-steroidal anti-inflammatory drug, flufenamic acid”. Bioorg. Med. Chem.7 (7): 1339–47. (July 1999). doi:10.1016/S0968-0896(99)00066-8. PMID10465408.
“The pathway by which the tetrameric protein transthyretin dissociates”. Biochemistry44 (47): 15525–33. (November 2005). doi:10.1021/bi051608t. PMID16300401.
“A peculiar form of peripheral neuropathy; familiar atypical generalized amyloidosis with special involvement of the peripheral nerves”. Brain75 (3): 408–27. (September 1952). doi:10.1093/brain/75.3.408. PMID12978172.
“Variant-sequence transthyretin (isoleucine 122) in late-onset cardiac amyloidosis in black Americans”. N. Engl. J. Med.336 (7): 466–73. (February 1997). doi:10.1056/NEJM199702133360703. PMID9017939.
“Partial denaturation of transthyretin is sufficient for amyloid fibril formation in vitro”. Biochemistry31 (36): 8654–60. (September 1992). doi:10.1021/bi00151a036. PMID1390650.
“The acid-mediated denaturation pathway of transthyretin yields a conformational intermediate that can self-assemble into amyloid”. Biochemistry35 (20): 6470–82. (May 1996). doi:10.1021/bi952501g. PMID8639594.
“Prevention of transthyretin amyloid disease by changing protein misfolding energetics”. Science299 (5607): 713–6. (January 2003). doi:10.1126/science.1079589. PMID12560553.
“An engineered transthyretin monomer that is nonamyloidogenic, unless it is partially denatured”. Biochemistry40 (38): 11442–52. (September 2001). doi:10.1021/bi011194d. PMID11560492.
臨床試験番号 NCT01960348 研究名 "APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis" - ClinicalTrials.gov