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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).
Zaidman CM, Proud CM, McDonald CM, Lehman KJ, Goedeker NL, Mason S, Murphy AP, Guridi M, Wang S, Reid C, Darton E, Wandel C, Lewis S, Malhotra J, Griffin DA, Potter RA, Rodino-Klapac LR, Mendell JR. Zaidman CM, et al. Among authors: wandel c. Ann Neurol. 2023 Nov;94(5):955-968. doi: 10.1002/ana.26755. Epub 2023 Sep 7. Ann Neurol. 2023. PMID: 37539981
AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial.
Mendell JR, Muntoni F, McDonald CM, Mercuri EM, Ciafaloni E, Komaki H, Leon-Astudillo C, Nascimento A, Proud C, Schara-Schmidt U, Veerapandiyan A, Zaidman CM, Guridi M, Murphy AP, Reid C, Wandel C, Asher DR, Darton E, Mason S, Potter RA, Singh T, Zhang W, Fontoura P, Elkins JS, Rodino-Klapac LR. Mendell JR, et al. Among authors: wandel c. Nat Med. 2024 Oct 9. doi: 10.1038/s41591-024-03304-z. Online ahead of print. Nat Med. 2024. PMID: 39385046
Balovaptan vs Placebo for Social Communication in Childhood Autism Spectrum Disorder: A Randomized Clinical Trial.
Hollander E, Jacob S, Jou R, McNamara N, Sikich L, Tobe R, Smith J, Sanders K, Squassante L, Murtagh L, Gleissl T, Wandel C, Veenstra-VanderWeele J. Hollander E, et al. Among authors: wandel c. JAMA Psychiatry. 2022 Aug 1;79(8):760-769. doi: 10.1001/jamapsychiatry.2022.1717. JAMA Psychiatry. 2022. PMID: 35793101 Free PMC article. Clinical Trial.
Efficacy and safety of balovaptan for socialisation and communication difficulties in autistic adults in North America and Europe: a phase 3, randomised, placebo-controlled trial.
Jacob S, Veenstra-VanderWeele J, Murphy D, McCracken J, Smith J, Sanders K, Meyenberg C, Wiese T, Deol-Bhullar G, Wandel C, Ashford E, Anagnostou E. Jacob S, et al. Among authors: wandel c. Lancet Psychiatry. 2022 Mar;9(3):199-210. doi: 10.1016/S2215-0366(21)00429-6. Epub 2022 Feb 10. Lancet Psychiatry. 2022. PMID: 35151410 Clinical Trial.
A randomized, double-blind, placebo-controlled phase II trial to explore the effects of a GABAA-α5 NAM (basmisanil) on intellectual disability associated with Down syndrome.
Goeldner C, Kishnani PS, Skotko BG, Casero JL, Hipp JF, Derks M, Hernandez MC, Khwaja O, Lennon-Chrimes S, Noeldeke J, Pellicer S, Squassante L, Visootsak J, Wandel C, Fontoura P, d'Ardhuy XL; Clematis Study Group. Goeldner C, et al. Among authors: wandel c. J Neurodev Disord. 2022 Feb 5;14(1):10. doi: 10.1186/s11689-022-09418-0. J Neurodev Disord. 2022. PMID: 35123401 Free PMC article. Clinical Trial.
Basmisanil, a highly selective GABAA-α5 negative allosteric modulator: preclinical pharmacology and demonstration of functional target engagement in man.
Hipp JF, Knoflach F, Comley R, Ballard TM, Honer M, Trube G, Gasser R, Prinssen E, Wallace TL, Rothfuss A, Knust H, Lennon-Chrimes S, Derks M, Bentley D, Squassante L, Nave S, Nöldeke J, Wandel C, Thomas AW, Hernandez MC. Hipp JF, et al. Among authors: wandel c. Sci Rep. 2021 Apr 8;11(1):7700. doi: 10.1038/s41598-021-87307-7. Sci Rep. 2021. PMID: 33833333 Free PMC article.
39 results