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Author

Elizabeth Maczek

Observed names: E. Maczek, Maczek E, Maczek, Elizabeth
Institution: Harvard University
ORCID: 0009-0001-1880-0482
Observed ORCID: 0009-0001-1880-0482
OpenAlex ID: A5073767177
Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial
2022 · Riccardo Masson, Maria Mazurkiewicz-Bełdzińska, et al. · The Lancet Neurology · 117
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Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study
2023 · Claudia A. Chiriboga, Claudio Bruno, et al. · Neurology and Therapy · 52
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The Impact of Erythropoietin on Short- and Long-Term Kidney-Related Outcomes in Neonates of Extremely Low Gestational Age. Results of a Multicenter, Double-Blind, Placebo-Controlled Randomized Clinical Trial
2021 · David J. Askenazi, Patrick J. Heagerty, et al. · The Journal of Pediatrics · 30
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Assessing Bulbar Function in Spinal Muscular Atrophy Using Patient-Reported Outcomes
2023 · Sally Dunaway Young, Amy Pasternak, et al. · Journal of Neuromuscular Diseases · 18
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P.47 Adaptive test for neuromuscular disorders: Design of a wheelchair-based assessment
2022 · T. Duong, W. Tang, et al. · Neuromuscular Disorders · 4
194P Longitudinal assessment of the Adapted Test for Neuromuscular Disease (ATEND) in individuals living with spinal muscular atrophy (SMA)
2024 · T. Duong, R. Muni-Lofra, et al. · Neuromuscular Disorders · 3
Association Between Urinary Biomarkers and CKD in Extremely Low Gestational Age Neonates
2023 · Sangeeta R. Hingorani, Robert H. Schmicker, et al. · American Journal of Kidney Diseases · 3
Deramiocel heart-derived cellular therapy in advanced Duchenne muscular dystrophy (HOPE-3): a phase 3, randomised, double-blind, placebo-controlled trial
2026 · Craig M McDonald, Chet Villa, et al. · The Lancet · 2
Efficacy and safety of risdiplam in patients with type 1 spinal muscular atrophy: a 3-year open-label extension of the two-part, phase 2 FIREFISH trial
2026 · Maria Mazurkiewicz-Bełdzińska, Maria Mazurkiewicz-Bełdzińska, et al. · The Lancet Child & Adolescent Health · 2
Correction: Serial casting for contractures in SMA: consensus derived guidelines for treatment
2025 · Laurey Brown, Katie Hoffman, et al. · Frontiers in Neurology · 2
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Sma: Registries, Biomarkers & Outcome Measures
2020 · T. Duong, A. Pasternak, et al. · Neuromuscular Disorders · 2
Correction to: Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study
2023 · Claudia A. Chiriboga, Claudio Bruno, et al. · Neurology and Therapy · 1
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Navigating new motor function trajectories: Consensus recommendations for assessment in the era of newborn screening and early treatment in SMA
2026 · Kristin J Krosschell, Sally Dunaway Young, et al. · Journal of Neuromuscular Diseases
Serial casting for contractures in SMA: consensus derived guidelines for treatment
2025 · Laurey Brown, Katie Hoffman, et al. · Frontiers in Neurology
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Serial casting for contractures in SMA: consensus derived guidelines for treatment
2025 · Brown L, Hoffman K, et al. · Newcastle University ePrints (Newcastle Univesity)
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138P Long-term motor responses to disease-modifying therapies in spinal muscular atrophy (SMA) adults: a prospective study
2024 · T. Duong, W. Tang, et al. · Neuromuscular Disorders
VP.13 Updates on the development of the spinal muscular atrophy - person-reported outcome (SMA-PRO): A caregiver and self-proxy performance measure for child
2022 · A. Pasternak, E. Maczek, et al. · Neuromuscular Disorders
Works count: 18
Citation count: 236
H-index: 4
I10-index: 4
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