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Page 1
Effects of oral sepiapterin on blood Phe concentration in a broad range of patients with phenylketonuria (APHENITY): results of an international, phase 3, randomised, double-blind, placebo-controlled trial.
Muntau AC, Longo N, Ezgu F, Schwartz IVD, Lah M, Bratkovic D, Margvelashvili L, Kiykim E, Zori R, Campistol Plana J, Bélanger-Quintana A, Lund A, Guilder L, Chakrapani A, Mungan HN, Guimas A, Cabrales Guerra IDC, MacDonald A, Ingalls K, Smith N; APHENITY study group. Muntau AC, et al. Among authors: bratkovic d. Lancet. 2024 Oct 5;404(10460):1333-1345. doi: 10.1016/S0140-6736(24)01556-3. Lancet. 2024. PMID: 39368841 Clinical Trial.
Correction: Long-term safety and efficacy of cipaglucosidase alfa plus miglustat in individuals living with Pompe disease: an open-label phase I/II study (ATB200-02).
Byrne BJ, Schoser B, Kishnani PS, Bratkovic D, Clemens PR, Goker-Alpan O, Ming X, Roberts M, Vorgerd M, Sivakumar K, van der Ploeg AT, Goldman M, Wright J, Holdbrook F, Jain V, Benjamin ER, Johnson F, Das SS, Wasfi Y, Mozaffar T. Byrne BJ, et al. Among authors: bratkovic d. J Neurol. 2025 Feb 17;272(3):213. doi: 10.1007/s00415-025-12899-3. J Neurol. 2025. PMID: 39960520 Free PMC article. No abstract available.
Exploring Quality of Life in Adults Living With Late-onset Pompe Disease: A Combined Quantitative and Qualitative Analysis of Patient Perceptions from Australia, France, Italy, and the Netherlands.
Lumgair H, Bashorum L, MacCulloch A, Minas E, Timmins G, Bratkovic D, Perry R, Stone M, Blazos V, Conti E, Saich R. Lumgair H, et al. Among authors: bratkovic d. J Health Econ Outcomes Res. 2025 Jan 2;12(1):1-12. doi: 10.36469/001c.126018. eCollection 2025. J Health Econ Outcomes Res. 2025. PMID: 39758431 Free PMC article.
104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease: a phase III open-label extension study (ATB200-07).
Schoser B, Kishnani PS, Bratkovic D, Byrne BJ, Claeys KG, Díaz-Manera J, Laforêt P, Roberts M, Toscano A, van der Ploeg AT, Castelli J, Goldman M, Holdbrook F, Sitaraman Das S, Wasfi Y, Mozaffar T; ATB200-07 Study Group. Schoser B, et al. Among authors: bratkovic d. J Neurol. 2024 May;271(5):2810-2823. doi: 10.1007/s00415-024-12236-0. Epub 2024 Feb 28. J Neurol. 2024. PMID: 38418563 Free PMC article. Clinical Trial.
Long-term safety and efficacy of cipaglucosidase alfa plus miglustat in individuals living with Pompe disease: an open-label phase I/II study (ATB200-02).
Byrne BJ, Schoser B, Kishnani PS, Bratkovic D, Clemens PR, Goker-Alpan O, Ming X, Roberts M, Vorgerd M, Sivakumar K, van der Ploeg AT, Goldman M, Wright J, Holdbrook F, Jain V, Benjamin ER, Johnson F, Das SS, Wasfi Y, Mozaffar T. Byrne BJ, et al. Among authors: bratkovic d. J Neurol. 2024 Apr;271(4):1787-1801. doi: 10.1007/s00415-023-12096-0. Epub 2023 Dec 6. J Neurol. 2024. PMID: 38057636 Free PMC article. Clinical Trial.
Intrathecal idursulfase-IT in children younger than 3 years with neuronopathic mucopolysaccharidosis II in a single-arm, open-label, phase 2/3 substudy and extension.
Muenzer J, Burton BK, Harmatz P, Gutiérrez-Solana LG, Ruiz-Garcia M, Jones SA, Guffon N, Inbar-Feigenberg M, Bratkovic D, Rust S, Hale M, Wu Y, Yee KS, Whiteman DAH, Alexanderian D. Muenzer J, et al. Among authors: bratkovic d. JIMD Rep. 2026 Feb 22;67(2):e12443. doi: 10.1002/jmd2.12443. eCollection 2026 Mar. JIMD Rep. 2026. PMID: 41737901 Free PMC article.
Switching Enzyme Replacement Therapy for Late-Onset Pompe Disease From Alglucosidase Alfa to Cipaglucosidase Alfa Plus Miglustat: Post Hoc Effect Size Analysis of PROPEL.
Kushlaf H, Díaz-Manera J, Bratkovic D, Byrne BJ, Claeys KG, Clemens PR, Dimachkie MM, Kishnani PS, Laforêt P, Roberts M, Schoser B, Toscano A, Castelli J, Holdbrook F, Sitaraman Das S, Goldman M, Mozaffar T; PROPEL Study Group. Kushlaf H, et al. Among authors: bratkovic d. Muscle Nerve. 2025 Aug;72(2):230-239. doi: 10.1002/mus.28420. Epub 2025 May 7. Muscle Nerve. 2025. PMID: 40342075 Clinical Trial.
Evaluation of early treatment with intravenous idursulfase and intrathecal idursulfase-IT on cognitive function in siblings with neuronopathic mucopolysaccharidosis II.
Muenzer J, Burton BK, Harmatz P, Gutiérrez-Solana LG, Ruiz-Garcia M, Jones SA, Guffon N, Inbar-Feigenberg M, Bratkovic D, Rust S, Hale M, Wu Y, Yee KS, Whiteman DAH, Alexanderian D. Muenzer J, et al. Among authors: bratkovic d. J Inherit Metab Dis. 2025 May;48(3):e12790. doi: 10.1002/jimd.12790. Epub 2024 Sep 9. J Inherit Metab Dis. 2025. PMID: 39252529 Free PMC article. Clinical Trial.
NewbornsInSA multi-omic newborn screening: protocol for a prospective cohort study.
Anastasi L, Chowdhury A, Ashenden A, Rozek T, Louise J, Lam K, Skinner S, Stallard K, Merlin T, Schubert C, Vogan A, Morris S, Cohen-Woods S, Smith N, Ranieri E, Saxon B, Scott HS, Bratkovic D, Barnett CP, Siu CW, King J, Kassahn KS. Anastasi L, et al. Among authors: bratkovic d. BMJ Open. 2026 Mar 11;16(3):e108406. doi: 10.1136/bmjopen-2025-108406. BMJ Open. 2026. PMID: 41813051 Free PMC article.
55 results