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Table representation of search results timeline featuring number of search results per year.

Year Number of Results
1993 3
2006 2
2007 1
2008 2
2009 1
2010 5
2011 5
2012 4
2013 11
2014 6
2015 14
2016 12
2017 12
2018 12
2019 18
2020 18
2021 21
2022 16
2023 21
2024 7

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151 results

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Page 1
Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria.
Balwani M, Sardh E, Ventura P, Peiró PA, Rees DC, Stölzel U, Bissell DM, Bonkovsky HL, Windyga J, Anderson KE, Parker C, Silver SM, Keel SB, Wang JD, Stein PE, Harper P, Vassiliou D, Wang B, Phillips J, Ivanova A, Langendonk JG, Kauppinen R, Minder E, Horie Y, Penz C, Chen J, Liu S, Ko JJ, Sweetser MT, Garg P, Vaishnaw A, Kim JB, Simon AR, Gouya L; ENVISION Investigators. Balwani M, et al. N Engl J Med. 2020 Jun 11;382(24):2289-2301. doi: 10.1056/NEJMoa1913147. N Engl J Med. 2020. PMID: 32521132 Clinical Trial.
Dersimelagon in Erythropoietic Protoporphyrias.
Balwani M, Bonkovsky HL, Levy C, Anderson KE, Bissell DM, Parker C, Takahashi F, Desnick RJ, Belongie K; Endeavor Investigators. Balwani M, et al. N Engl J Med. 2023 Apr 13;388(15):1376-1385. doi: 10.1056/NEJMoa2208754. N Engl J Med. 2023. PMID: 37043653 Clinical Trial.
X-Linked Protoporphyria.
Balwani M, Desnick R; Porphyrias Consortium of the NIH-Sponsored Rare Diseases Clinical Research Network. Balwani M, et al. 2013 Feb 14 [updated 2019 Nov 27]. In: Adam MP, Feldman J, Mirzaa GM, Pagon RA, Wallace SE, Bean LJH, Gripp KW, Amemiya A, editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993–2024. 2013 Feb 14 [updated 2019 Nov 27]. In: Adam MP, Feldman J, Mirzaa GM, Pagon RA, Wallace SE, Bean LJH, Gripp KW, Amemiya A, editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993–2024. PMID: 23409301 Free Books & Documents. Review.
Update on the Porphyrias.
Dickey AK, Leaf RK, Balwani M. Dickey AK, et al. Among authors: balwani m. Annu Rev Med. 2024 Jan 29;75:321-335. doi: 10.1146/annurev-med-042921-123602. Epub 2023 Aug 4. Annu Rev Med. 2024. PMID: 37540847 Free article. Review.
RNA interference therapy in acute hepatic porphyrias.
Yasuda M, Keel S, Balwani M. Yasuda M, et al. Among authors: balwani m. Blood. 2023 Nov 9;142(19):1589-1599. doi: 10.1182/blood.2022018662. Blood. 2023. PMID: 37027823 Free article.
Congenital Erythropoietic Porphyria.
Erwin A, Balwani M, Desnick RJ; Porphyrias Consortium of the NIH-Sponsored Rare Diseases Clinical Research Network. Erwin A, et al. Among authors: balwani m. 2013 Sep 12 [updated 2021 Apr 15]. In: Adam MP, Feldman J, Mirzaa GM, Pagon RA, Wallace SE, Bean LJH, Gripp KW, Amemiya A, editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993–2024. 2013 Sep 12 [updated 2021 Apr 15]. In: Adam MP, Feldman J, Mirzaa GM, Pagon RA, Wallace SE, Bean LJH, Gripp KW, Amemiya A, editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993–2024. PMID: 24027798 Free Books & Documents. Review.
Reply.
Wang B, Balwani M, Bonkovsky HL, Anderson KE, Bloomer JR, Bissell DM, Phillips JD, Desnick RJ; Porphyrias Consortium of the Rare Diseases Clinical Research Network. Wang B, et al. Among authors: balwani m. Hepatology. 2018 Feb;67(2):803-804. doi: 10.1002/hep.29674. Epub 2018 Jan 2. Hepatology. 2018. PMID: 29159969 No abstract available.
Porphyrias in the Age of Targeted Therapies.
Erwin AL, Balwani M. Erwin AL, et al. Among authors: balwani m. Diagnostics (Basel). 2021 Sep 29;11(10):1795. doi: 10.3390/diagnostics11101795. Diagnostics (Basel). 2021. PMID: 34679493 Free PMC article. Review.
Diagnostic Delay in Erythropoietic Protoporphyria.
Lala SM, Naik H, Balwani M. Lala SM, et al. Among authors: balwani m. J Pediatr. 2018 Nov;202:320-323.e2. doi: 10.1016/j.jpeds.2018.06.001. Epub 2018 Jul 2. J Pediatr. 2018. PMID: 30041937 Free PMC article. Review.
151 results