Genetisch-basierte Therapien bei Muskelkrankheiten
Author:
Publisher
Springer Science and Business Media LLC
Subject
General Medicine
Link
https://link.springer.com/content/pdf/10.1007/s15005-023-3509-z.pdf
Reference24 articles.
1. Brakemeier S, Stolte B, Kleinschnitz C, Hagenacker T. Treatment of Adult Spinal Muscular Atrophy: Overview and Recent Developments. Curr Pharm Des. 2022;28(11):892-8
2. Meijboom KE, Brown RH. Approaches to Gene Modulation Therapy for ALS. Neurotherapeutics. 2022;19(4):1159-79
3. Zaidman CM, Proud CM, McDonald CM et al. Delandistrogene Moxeparvovec Gene Therapy in Ambulatory Patients (Aged >/=4 to <8 Years) with Duchenne Muscular Dystrophy: 1-Year Interim Results from Study SRP-9001-103 (ENDEAVOR). Ann Neurol. 2023;94(5):955-68
4. Whiteley LO. An Overview of Nonclinical and Clinical Liver Toxicity Associated With AAV Gene Therapy. Toxicol Pathol. 2023:1926233231201408
5. Chand D, Mohr F, McMillan H et al. Hepatotoxicity following administration of onasemnogene abeparvovec (AVXS-101) for the treatment of spinal muscular atrophy. J Hepatol. 2021;74(3):560-6
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