Clinical trial number NCT01960348 for "APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis" at ClinicalTrials.gov
doi.org
Razavi H, Palaninathan SK, Powers ET, Wiseman RL, Purkey HE, Mohamedmohaideen NN, Deechongkit S, Chiang KP, Dendle MT, Sacchettini JC, Kelly JW (June 2003). "Benzoxazoles as transthyretin amyloid fibril inhibitors: synthesis, evaluation, and mechanism of action". Angew. Chem. Int. Ed. Engl. 42 (24): 2758–61. doi:10.1002/anie.200351179. PMID12820260.
Sekijima Y, Dendle MA, Kelly JW (December 2006). "Orally administered diflunisal stabilizes transthyretin against dissociation required for amyloidogenesis". Amyloid. 13 (4): 236–49. doi:10.1080/13506120600960882. PMID17107884. S2CID32736564.
Adamski-Werner SL, Palaninathan SK, Sacchettini JC, Kelly JW (January 2004). "Diflunisal analogues stabilize the native state of transthyretin. Potent inhibition of amyloidogenesis". J. Med. Chem. 47 (2): 355–74. 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". In Seldin DC, Skinner M, Berk JL, Connors LH (eds.). XIth International Symposium on Amyloidosis. Boca Raton: CRC. pp. 205–207. doi:10.1201/9781420043358.ch69. ISBN978-1-4200-4281-8.
Baures PW, Oza VB, Peterson SA, Kelly JW (July 1999). "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. doi:10.1016/S0968-0896(99)00066-8. PMID10465408.
Foss TR, Wiseman RL, Kelly JW (November 2005). "The pathway by which the tetrameric protein transthyretin dissociates". Biochemistry. 44 (47): 15525–33. doi:10.1021/bi051608t. PMID16300401.
Andrade C (September 1952). "A peculiar form of peripheral neuropathy; familiar atypical generalized amyloidosis with special involvement of the peripheral nerves". Brain. 75 (3): 408–27. doi:10.1093/brain/75.3.408. PMID12978172.
Colon W, Kelly JW (September 1992). "Partial denaturation of transthyretin is sufficient for amyloid fibril formation in vitro". Biochemistry. 31 (36): 8654–60. doi:10.1021/bi00151a036. PMID1390650.
Lai Z, Colón W, Kelly JW (May 1996). "The acid-mediated denaturation pathway of transthyretin yields a conformational intermediate that can self-assemble into amyloid". Biochemistry. 35 (20): 6470–82. doi:10.1021/bi952501g. PMID8639594.
Jiang X, Smith CS, Petrassi HM, Hammarström P, White JT, Sacchettini JC, Kelly JW (September 2001). "An engineered transthyretin monomer that is nonamyloidogenic, unless it is partially denatured". Biochemistry. 40 (38): 11442–52. doi:10.1021/bi011194d. PMID11560492.
Razavi H, Palaninathan SK, Powers ET, Wiseman RL, Purkey HE, Mohamedmohaideen NN, Deechongkit S, Chiang KP, Dendle MT, Sacchettini JC, Kelly JW (June 2003). "Benzoxazoles as transthyretin amyloid fibril inhibitors: synthesis, evaluation, and mechanism of action". Angew. Chem. Int. Ed. Engl. 42 (24): 2758–61. doi:10.1002/anie.200351179. PMID12820260.
Sekijima Y, Dendle MA, Kelly JW (December 2006). "Orally administered diflunisal stabilizes transthyretin against dissociation required for amyloidogenesis". Amyloid. 13 (4): 236–49. doi:10.1080/13506120600960882. PMID17107884. S2CID32736564.
Adamski-Werner SL, Palaninathan SK, Sacchettini JC, Kelly JW (January 2004). "Diflunisal analogues stabilize the native state of transthyretin. Potent inhibition of amyloidogenesis". J. Med. Chem. 47 (2): 355–74. doi:10.1021/jm030347n. PMID14711308.
Baures PW, Oza VB, Peterson SA, Kelly JW (July 1999). "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. doi:10.1016/S0968-0896(99)00066-8. PMID10465408.
Foss TR, Wiseman RL, Kelly JW (November 2005). "The pathway by which the tetrameric protein transthyretin dissociates". Biochemistry. 44 (47): 15525–33. doi:10.1021/bi051608t. PMID16300401.
Andrade C (September 1952). "A peculiar form of peripheral neuropathy; familiar atypical generalized amyloidosis with special involvement of the peripheral nerves". Brain. 75 (3): 408–27. doi:10.1093/brain/75.3.408. PMID12978172.
Colon W, Kelly JW (September 1992). "Partial denaturation of transthyretin is sufficient for amyloid fibril formation in vitro". Biochemistry. 31 (36): 8654–60. doi:10.1021/bi00151a036. PMID1390650.
Lai Z, Colón W, Kelly JW (May 1996). "The acid-mediated denaturation pathway of transthyretin yields a conformational intermediate that can self-assemble into amyloid". Biochemistry. 35 (20): 6470–82. doi:10.1021/bi952501g. PMID8639594.
Jiang X, Smith CS, Petrassi HM, Hammarström P, White JT, Sacchettini JC, Kelly JW (September 2001). "An engineered transthyretin monomer that is nonamyloidogenic, unless it is partially denatured". Biochemistry. 40 (38): 11442–52. doi:10.1021/bi011194d. PMID11560492.