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

Year Number of Results
2002 1
2004 1
2005 2
2006 2
2009 1
2010 1
2011 5
2012 7
2013 5
2014 3
2015 6
2016 7
2017 6
2018 4
2019 9
2020 11
2021 5
2022 8
2023 4
2024 2

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75 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. Among authors: langendonk jg. 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.
Barman-Aksözen J, Langendonk JG. Barman-Aksözen J, et al. Among authors: langendonk jg. N Engl J Med. 2023 Jun 29;388(26):2492. doi: 10.1056/NEJMc2305601. N Engl J Med. 2023. PMID: 37379150 No abstract available.
Fertility in adult women with classic galactosemia and primary ovarian insufficiency.
van Erven B, Berry GT, Cassiman D, Connolly G, Forga M, Gautschi M, Gubbels CS, Hollak CEM, Janssen MC, Knerr I, Labrune P, Langendonk JG, Õunap K, Thijs A, Vos R, Wortmann SB, Rubio-Gozalbo ME. van Erven B, et al. Among authors: langendonk jg. Fertil Steril. 2017 Jul;108(1):168-174. doi: 10.1016/j.fertnstert.2017.05.013. Epub 2017 Jun 1. Fertil Steril. 2017. PMID: 28579413 Free article. Review.
Liver involvement in patients with erythropoietic protoporphyria.
Wensink D, Coenen S, Wilson JHP, Wagenmakers MAEM, Langendonk JG. Wensink D, et al. Among authors: langendonk jg. Dig Liver Dis. 2022 Apr;54(4):515-520. doi: 10.1016/j.dld.2021.08.007. Epub 2021 Aug 30. Dig Liver Dis. 2022. PMID: 34475006 Free article.
Afamelanotide for Erythropoietic Protoporphyria.
Langendonk JG, Balwani M, Anderson KE, Bonkovsky HL, Anstey AV, Bissell DM, Bloomer J, Edwards C, Neumann NJ, Parker C, Phillips JD, Lim HW, Hamzavi I, Deybach JC, Kauppinen R, Rhodes LE, Frank J, Murphy GM, Karstens FPJ, Sijbrands EJG, de Rooij FWM, Lebwohl M, Naik H, Goding CR, Wilson JHP, Desnick RJ. Langendonk JG, et al. N Engl J Med. 2015 Jul 2;373(1):48-59. doi: 10.1056/NEJMoa1411481. N Engl J Med. 2015. PMID: 26132941 Free PMC article. Clinical Trial.
Primary carnitine deficiency is a life-long disease.
Crefcoeur LL, Melles MC, Bruning TA, Pereira RR, Langendonk JG. Crefcoeur LL, et al. Among authors: langendonk jg. JIMD Rep. 2022 Jul 27;63(6):524-528. doi: 10.1002/jmd2.12319. eCollection 2022 Nov. JIMD Rep. 2022. PMID: 36341172 Free PMC article.
Acute intermittent porphyria-related leukoencephalopathy.
Kevelam SH, Neeleman RA, Waisfisz Q, Friesema EC, Langendonk JG, van der Knaap MS. Kevelam SH, et al. Among authors: langendonk jg. Neurology. 2016 Sep 20;87(12):1258-65. doi: 10.1212/WNL.0000000000003129. Epub 2016 Aug 24. Neurology. 2016. PMID: 27558376
75 results