Difference between revisions of "Verbrinacogene setparvovec (FLT-180a)"
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(Created page with "Gene therapy ==Preliminary data== ===Hemophilia B=== #'''B-AMAZE:''' Chowdary P, Shapiro S, Makris M, Evans G, Boyce S, Talks K, Dolan G, Reiss U,...") |
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#'''B-AMAZE:''' Chowdary P, Shapiro S, Makris M, Evans G, Boyce S, Talks K, Dolan G, Reiss U, Phillips M, Riddell A, Peralta MR, Quaye M, Patch DW, Tuddenham E, Dane A, Watissée M, Long A, Nathwani A. Phase 1-2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B. N Engl J Med. 2022 Jul 21;387(3):237-247. [https://doi.org/10.1056/nejmoa2119913 link to original article] [https://pubmed.ncbi.nlm.nih.gov/35857660/ PubMed] NCT03369444; NCT03641703 | #'''B-AMAZE:''' Chowdary P, Shapiro S, Makris M, Evans G, Boyce S, Talks K, Dolan G, Reiss U, Phillips M, Riddell A, Peralta MR, Quaye M, Patch DW, Tuddenham E, Dane A, Watissée M, Long A, Nathwani A. Phase 1-2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B. N Engl J Med. 2022 Jul 21;387(3):237-247. [https://doi.org/10.1056/nejmoa2119913 link to original article] [https://pubmed.ncbi.nlm.nih.gov/35857660/ PubMed] NCT03369444; NCT03641703 | ||
| + | [[Category:Inherited coagulopathy medications (investigational)]] | ||
[[Category:Investigational drugs]] | [[Category:Investigational drugs]] | ||
[[Category:Stub]] | [[Category:Stub]] | ||
Revision as of 11:20, 2 September 2022
Gene therapy
Preliminary data
Hemophilia B
- B-AMAZE: Chowdary P, Shapiro S, Makris M, Evans G, Boyce S, Talks K, Dolan G, Reiss U, Phillips M, Riddell A, Peralta MR, Quaye M, Patch DW, Tuddenham E, Dane A, Watissée M, Long A, Nathwani A. Phase 1-2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B. N Engl J Med. 2022 Jul 21;387(3):237-247. link to original article PubMed NCT03369444; NCT03641703