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Page 1
CRISPR-Based Therapy for Hereditary Angioedema.
Cohn DM, Gurugama P, Magerl M, Katelaris CH, Launay D, Bouillet L, Petersen RS, Lindsay K, Aygören-Pürsün E, Maag D, Butler JS, Shah MY, Golden A, Xu Y, Abdelhady AM, Lebwohl D, Longhurst HJ. Cohn DM, et al. N Engl J Med. 2024 Oct 24. doi: 10.1056/NEJMoa2405734. Online ahead of print. N Engl J Med. 2024. PMID: 39445704
CRISPR-Cas9 In Vivo Gene Editing of KLKB1 for Hereditary Angioedema.
Longhurst HJ, Lindsay K, Petersen RS, Fijen LM, Gurugama P, Maag D, Butler JS, Shah MY, Golden A, Xu Y, Boiselle C, Vogel JD, Abdelhady AM, Maitland ML, McKee MD, Seitzer J, Han BW, Soukamneuth S, Leonard J, Sepp-Lorenzino L, Clark ED, Lebwohl D, Cohn DM. Longhurst HJ, et al. Among authors: cohn dm. N Engl J Med. 2024 Feb 1;390(5):432-441. doi: 10.1056/NEJMoa2309149. N Engl J Med. 2024. PMID: 38294975 Clinical Trial.
Oral Sebetralstat for On-Demand Treatment of Hereditary Angioedema Attacks.
Riedl MA, Farkas H, Aygören-Pürsün E, Psarros F, Soteres DF, Staevska M, Cancian M, Hagin D, Honda D, Melamed I, Savic S, Stobiecki M, Busse PJ, Dias de Castro E, Agmon-Levin N, Gower R, Kessel A, Kurowski M, Lleonart R, Grivcheva Panovska V, Wedner HJ, Audhya PK, Hao J, Iverson M, Smith MD, Yea CM, Lumry WR, Zanichelli A, Bernstein JA, Maurer M, Cohn DM; KONFIDENT Investigators. Riedl MA, et al. Among authors: cohn dm. N Engl J Med. 2024 Jul 4;391(1):32-43. doi: 10.1056/NEJMoa2314192. Epub 2024 May 31. N Engl J Med. 2024. PMID: 38819658 Clinical Trial.
The international WAO/EAACI guideline for the management of hereditary angioedema-The 2021 revision and update.
Maurer M, Magerl M, Betschel S, Aberer W, Ansotegui IJ, Aygören-Pürsün E, Banerji A, Bara NA, Boccon-Gibod I, Bork K, Bouillet L, Boysen HB, Brodszki N, Busse PJ, Bygum A, Caballero T, Cancian M, Castaldo A, Cohn DM, Csuka D, Farkas H, Gompels M, Gower R, Grumach AS, Guidos-Fogelbach G, Hide M, Kang HR, Kaplan AP, Katelaris C, Kiani-Alikhan S, Lei WT, Lockey R, Longhurst H, Lumry WR, MacGinnitie A, Malbran A, Martinez Saguer I, Matta JJ, Nast A, Nguyen D, Nieto-Martinez SA, Pawankar R, Peter J, Porebski G, Prior N, Reshef A, Riedl M, Ritchie B, Rafique Sheikh F, Smith WB, Spaeth PJ, Stobiecki M, Toubi E, Varga LA, Weller K, Zanichelli A, Zhi Y, Zuraw B, Craig T. Maurer M, et al. Among authors: cohn dm. Allergy. 2022 Jul;77(7):1961-1990. doi: 10.1111/all.15214. Epub 2022 Feb 3. Allergy. 2022. PMID: 35006617
The international WAO/EAACI guideline for the management of hereditary angioedema - The 2021 revision and update.
Maurer M, Magerl M, Betschel S, Aberer W, Ansotegui IJ, Aygören-Pürsün E, Banerji A, Bara NA, Boccon-Gibod I, Bork K, Bouillet L, Boysen HB, Brodszki N, Busse PJ, Bygum A, Caballero T, Cancian M, Castaldo AJ, Cohn DM, Csuka D, Farkas H, Gompels M, Gower R, Grumach AS, Guidos-Fogelbach G, Hide M, Kang HR, Kaplan AP, Katelaris CH, Kiani-Alikhan S, Lei WT, Lockey RF, Longhurst H, Lumry W, MacGinnitie A, Malbran A, Martinez Saguer I, Matta Campos JJ, Nast A, Nguyen D, Nieto-Martinez SA, Pawankar R, Peter J, Porebski G, Prior N, Reshef A, Riedl M, Ritchie B, Sheikh FR, Smith WB, Spaeth PJ, Stobiecki M, Toubi E, Varga LA, Weller K, Zanichelli A, Zhi Y, Zuraw B, Craig T. Maurer M, et al. Among authors: cohn dm. World Allergy Organ J. 2022 Apr 7;15(3):100627. doi: 10.1016/j.waojou.2022.100627. eCollection 2022 Mar. World Allergy Organ J. 2022. PMID: 35497649 Free PMC article.
An investigational oral plasma kallikrein inhibitor for on-demand treatment of hereditary angioedema: a two-part, randomised, double-blind, placebo-controlled, crossover phase 2 trial.
Aygören-Pürsün E, Zanichelli A, Cohn DM, Cancian M, Hakl R, Kinaciyan T, Magerl M, Martinez-Saguer I, Stobiecki M, Farkas H, Kiani-Alikhan S, Grivcheva-Panovska V, Bernstein JA, Li HH, Longhurst HJ, Audhya PK, Smith MD, Yea CM, Maetzel A, Lee DK, Feener EP, Gower R, Lumry WR, Banerji A, Riedl MA, Maurer M. Aygören-Pürsün E, et al. Among authors: cohn dm. Lancet. 2023 Feb 11;401(10375):458-469. doi: 10.1016/S0140-6736(22)02406-0. Lancet. 2023. PMID: 36774155 Clinical Trial.
A Core Outcome Set for Efficacy of Acute Treatment of Hereditary Angioedema.
Petersen RS, Fijen LM, Apfelbacher C, Magerl M, Weller K, Aberer W, Adatia A, Audhya P, Bara NA, Betschel S, Boccon-Gibod I, Bouillet L, Brodszki N, Busse PJ, Buttgereit T, Bygum A, Cancian M, Craig T, Csuka D, Farkas H, Fomina D, Gil-Serrano J, Gompels M, Guidos Fogelbach G, Guilarte M, Hide M, Kiani-Alikhan S, Kinaciyan T, Lenten A, Lleonart R, Longhurst H, Lumry WR, Malbran A, Malinauskiene L, Matta Campos JJ, Mendivil J, Nieto-Martinez SA, Peter JG, Porebski G, Reshef A, Riedl M, Valerieva A, Waserman S, Maurer M, Cohn DM. Petersen RS, et al. Among authors: cohn dm. J Allergy Clin Immunol Pract. 2024 Jun;12(6):1614-1621. doi: 10.1016/j.jaip.2024.04.007. Epub 2024 Apr 11. J Allergy Clin Immunol Pract. 2024. PMID: 38609017 Free article.
Efficacy and Safety of Donidalorsen for Hereditary Angioedema.
Riedl MA, Tachdjian R, Lumry WR, Craig T, Karakaya G, Gelincik A, Stobiecki M, Jacobs JS, Gokmen NM, Reshef A, Gompels MM, Manning ME, Bordone L, Newman KB, Treadwell S, Wang S, Yarlas A, Cohn DM; OASIS-HAE Team. Riedl MA, et al. Among authors: cohn dm. N Engl J Med. 2024 Jul 4;391(1):21-31. doi: 10.1056/NEJMoa2402478. Epub 2024 May 31. N Engl J Med. 2024. PMID: 38819395 Clinical Trial.
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