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Gene replacement therapy with onasemnogene abeparvovec in children with spinal muscular atrophy aged 24 months or younger and bodyweight up to 15 kg: an observational cohort study.
Weiß C, Ziegler A, Becker LL, Johannsen J, Brennenstuhl H, Schreiber G, Flotats-Bastardas M, Stoltenburg C, Hartmann H, Illsinger S, Denecke J, Pechmann A, Müller-Felber W, Vill K, Blaschek A, Smitka M, van der Stam L, Weiss K, Winter B, Goldhahn K, Plecko B, Horber V, Bernert G, Husain RA, Rauscher C, Trollmann R, Garbade SF, Hahn A, von der Hagen M, Kaindl AM. Weiß C, et al. Among authors: hahn a. Lancet Child Adolesc Health. 2022 Jan;6(1):17-27. doi: 10.1016/S2352-4642(21)00287-X. Epub 2021 Oct 29. Lancet Child Adolesc Health. 2022. PMID: 34756190
Diagnosis and Care of Infants and Children with Pompe Disease.
Hahn A, Hennermann JB, Huemer M, Kampmann C, Marquardt T, Mengel E, Müller-Felber W, Muschol N, Rohrbach M, Stehling F. Hahn A, et al. Klin Padiatr. 2020 Feb 18. doi: 10.1055/a-1110-7335. Online ahead of print. Klin Padiatr. 2020. PMID: 32069498 English.
[Recommendations for gene therapy of spinal muscular atrophy with onasemnogene abeparvovec-AVXS-101 : Consensus paper of the German representatives of the Society for Pediatric Neurology (GNP) and the German treatment centers with collaboration of the medical scientific advisory board of the German Society for Muscular Diseases (DGM)].
Ziegler A, Wilichowski E, Schara U, Hahn A, Müller-Felber W, Johannsen J, von der Hagen M, von Moers A, Stoltenburg C, Saffari A, Walter MC, Husain RA, Pechmann A, Köhler C, Horber V, Schwartz O, Kirschner J. Ziegler A, et al. Among authors: hahn a. Nervenarzt. 2020 Jun;91(6):518-529. doi: 10.1007/s00115-020-00919-8. Nervenarzt. 2020. PMID: 32394004 Review. German.
New Therapeutics Options for Pediatric Neuromuscular Disorders.
Flotats-Bastardas M, Hahn A. Flotats-Bastardas M, et al. Among authors: hahn a. Front Pediatr. 2020 Nov 23;8:583877. doi: 10.3389/fped.2020.583877. eCollection 2020. Front Pediatr. 2020. PMID: 33330280 Free PMC article. Review.
Prescription patterns of antiseizure drugs in tuberous sclerosis complex (TSC)-associated epilepsy: a multicenter cohort study from Germany and review of the literature.
Strzelczyk A, Grau J, Bast T, Bertsche A, Bettendorf U, Hahn A, Hartmann H, Hertzberg C, Hornemann F, Immisch I, Jacobs J, Klotz KA, Kluger G, Knake S, Knuf M, Kurlemann G, Marquard K, Mayer T, Meyer S, Muhle H, Müller-Schlüter K, von Podewils F, Rosenow F, Ruf S, Sauter M, Schäfer H, Schlump JU, Schubert-Bast S, Syrbe S, Thiels C, Trollmann R, Wiemer-Kruel A, Wilken B, Zukunft B, Zöllner JP. Strzelczyk A, et al. Among authors: hahn a. Expert Rev Clin Pharmacol. 2021 Jun;14(6):749-760. doi: 10.1080/17512433.2021.1911643. Epub 2021 May 31. Expert Rev Clin Pharmacol. 2021. PMID: 33792454 Review.
Direct and indirect costs and cost-driving factors of Tuberous sclerosis complex in children, adolescents, and caregivers: a multicenter cohort study.
Grau J, Zöllner JP, Schubert-Bast S, Kurlemann G, Hertzberg C, Wiemer-Kruel A, Bast T, Bertsche A, Bettendorf U, Fiedler B, Hahn A, Hartmann H, Hornemann F, Immisch I, Jacobs J, Kieslich M, Klein KM, Klotz KA, Kluger G, Knuf M, Mayer T, Marquard K, Meyer S, Muhle H, Müller-Schlüter K, Noda AH, Ruf S, Sauter M, Schlump JU, Syrbe S, Thiels C, Trollmann R, Wilken B, Willems LM, Rosenow F, Strzelczyk A. Grau J, et al. Among authors: hahn a. Orphanet J Rare Dis. 2021 Jun 21;16(1):282. doi: 10.1186/s13023-021-01899-x. Orphanet J Rare Dis. 2021. PMID: 34154622 Free PMC article.
1,852 results