Leigh Maria Ramos-Platt, MD

Clinical Professor of Neurology and Pediatrics (Clinician Educator)

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Publications

Deramiocel heart-derived cellular therapy in advanced Duchenne muscular dystrophy (HOPE-3): a phase 3, randomised, double-blind, placebo-controlled trial. Lancet. 2026 Jul 29.. View in PubMed
Effect of the COVID-19 Pandemic on the Quality of Life of Pediatric Patients with Myasthenia. J Child Neurol. 2026 Apr; 41(4):499-504.. View in PubMed
Consensus recommendations and considerations for the delivery and monitoring of gene therapy in patients with Duchenne muscular dystrophy. Neuromuscul Disord. 2025 Sep; 54:106208.. View in PubMed
Access to novel therapies for Duchenne muscular dystrophy-Insights from expert treating physicians. Ann Child Neurol Soc. 2024 Sep; 2(3):184-188.. View in PubMed
Safety and tolerability of intravenous immunoglobulin infusion in Down syndrome regression disorder. Am J Med Genet A. 2024 05; 194(5):e63524.. View in PubMed
Experience and Perspectives in the US on the Evolving Treatment Landscape in Spinal Muscular Atrophy. Int J Gen Med. 2022; 15:7341-7353.. View in PubMed
Early-Onset Late Gadolinium Enhancement is a Prognostic Factor for Duchenne Cardiomyopathy. Pediatr Cardiol. 2023 Feb; 44(2):433-440.. View in PubMed
Diffuse Anaplastic Wilms Tumor in a Child With LAMA2 -related Muscular Dystrophy. J Pediatr Hematol Oncol. 2022 11 01; 44(8):474-478.. View in PubMed
Does acute flaccid myelitis cause respiratory failure in children? Pediatr Pulmonol. 2022 03; 57(3):682-685. Lazzarini LM, Werner JM, Perez IA , Seruya M, Ramos-Platt L , Tiongson E , Keens TG . View in PubMed
Characteristics of Upper Extremity Recovery in Acute Flaccid Myelitis: A Case Series. Plast Reconstr Surg. 2021 03 01; 147(3):645-655.. View in PubMed