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Antisense oligonucleotide targeting DMPK in patients with myotonic dystrophy type 1: a multicentre, randomised, dose-escalation, placebo-controlled, phase 1/2a trial.
Thornton CA, Moxley RT 3rd, Eichinger K, Heatwole C, Mignon L, Arnold WD, Ashizawa T, Day JW, Dent G, Tanner MK, Duong T, Greene EP, Herbelin L, Johnson NE, King W, Kissel JT, Leung DG, Lott DJ, Norris DA, Pucillo EM, Schell W, Statland JM, Stinson N, Subramony SH, Xia S, Bishop KM, Bennett CF. Thornton CA, et al. Among authors: bishop km. Lancet Neurol. 2023 Mar;22(3):218-228. doi: 10.1016/S1474-4422(23)00001-7. Lancet Neurol. 2023. PMID: 36804094 Clinical Trial.
Trofinetide for the treatment of Rett syndrome: a randomized phase 3 study.
Neul JL, Percy AK, Benke TA, Berry-Kravis EM, Glaze DG, Marsh ED, Lin T, Stankovic S, Bishop KM, Youakim JM. Neul JL, et al. Among authors: bishop km. Nat Med. 2023 Jun;29(6):1468-1475. doi: 10.1038/s41591-023-02398-1. Epub 2023 Jun 8. Nat Med. 2023. PMID: 37291210 Free PMC article. Clinical Trial.
Development of trofinetide for the treatment of Rett syndrome: from bench to bedside.
Kennedy M, Glass L, Glaze DG, Kaminsky S, Percy AK, Neul JL, Jones NE, Tropea D, Horrigan JP, Nues P, Bishop KM, Youakim JM. Kennedy M, et al. Among authors: bishop km. Front Pharmacol. 2024 Jan 22;14:1341746. doi: 10.3389/fphar.2023.1341746. eCollection 2023. Front Pharmacol. 2024. PMID: 38318312 Free PMC article.
Treatment of infantile-onset spinal muscular atrophy with nusinersen: final report of a phase 2, open-label, multicentre, dose-escalation study.
Finkel RS, Chiriboga CA, Vajsar J, Day JW, Montes J, De Vivo DC, Bishop KM, Foster R, Liu Y, Ramirez-Schrempp D, Schneider E, Bennett CF, Wong J, Farwell W. Finkel RS, et al. Among authors: bishop km. Lancet Child Adolesc Health. 2021 Jul;5(7):491-500. doi: 10.1016/S2352-4642(21)00100-0. Epub 2021 Jun 3. Lancet Child Adolesc Health. 2021. PMID: 34089650 Clinical Trial.
Nusinersen improves walking distance and reduces fatigue in later-onset spinal muscular atrophy.
Montes J, Dunaway Young S, Mazzone ES, Pasternak A, Glanzman AM, Finkel RS, Darras BT, Muntoni F, Mercuri E, De Vivo DC, Bishop KM, Schneider E, Bennett CF, Foster R, Farwell W; CS2 and CS12 Study Groups. Montes J, et al. Among authors: bishop km. Muscle Nerve. 2019 Oct;60(4):409-414. doi: 10.1002/mus.26633. Epub 2019 Jul 27. Muscle Nerve. 2019. PMID: 31298747 Free PMC article. Clinical Trial.
40 results