Michelle S. Lorentzos

Westmead, NSW, Australia

Last Updated: 09/26/2026

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Found 16 publications

Early brain-penetrant immunotherapy reverses interferon signature and improves motor outcome in a case of ADAR1-related Aicardi-Goutières syndrome.

Early brain-penetrant immunotherapy reverses interferon signature and improves motor outcome in a case of ADAR1-related Aicardi-Goutières syndrome.

Journal: Clinical & translational immunology

Published: April 16, 2026

Strategic consensus on the clinical translation of advanced therapies in paediatric rare neurological disorders.

Strategic consensus on the clinical translation of advanced therapies in paediatric rare neurological disorders.

Journal: Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics

Published: February 05, 2026

cGAS inhibitor IMSB301 modifies interferon signalling in peripheral mononuclear cells of SAMHD1 genetic interferonopathy in vitro.

cGAS inhibitor IMSB301 modifies interferon signalling in peripheral mononuclear cells of SAMHD1 genetic interferonopathy in vitro.

Journal: Clinical & translational immunology

Published: December 14, 2025

Confirmatory long-term efficacy and safety results of ataluren in patients with nmDMD from Study 041, an international, randomized, double-blind, placebo-controlled, Phase III trial.

Confirmatory long-term efficacy and safety results of ataluren in patients with nmDMD from Study 041, an international, randomized, double-blind, placebo-controlled, Phase III trial.

Journal: Journal of comparative effectiveness research

Published: August 22, 2025

Early diagnosis of Duchenne muscular dystrophy - A Treat-NMD international workshop.

Early diagnosis of Duchenne muscular dystrophy - A Treat-NMD international workshop.

Journal: Neuromuscular disorders : NMD

Published: September 26, 2024

Newborn screening for Duchenne muscular dystrophy: the perspectives of stakeholders.

Newborn screening for Duchenne muscular dystrophy: the perspectives of stakeholders.

Journal: The Lancet regional health. Western Pacific

Published: November 19, 2023

Mechanical Thrombectomy for Pediatric Arterial Ischemic Stroke from Acute M2 Occlusion.

Mechanical Thrombectomy for Pediatric Arterial Ischemic Stroke from Acute M2 Occlusion.

Journal: AJNR. American journal of neuroradiology

Published: October 06, 2023

Providing Australian children and adolescents with equitable access to new and emerging therapies through clinical trials: a call to action.

Providing Australian children and adolescents with equitable access to new and emerging therapies through clinical trials: a call to action.

Journal: The Medical journal of Australia

Published: April 13, 2023

Newborn Screening for the Diagnosis and Treatment of Duchenne Muscular Dystrophy.

Newborn Screening for the Diagnosis and Treatment of Duchenne Muscular Dystrophy.

Journal: Journal of neuromuscular diseases

Published: November 14, 2022

Rapid onset functional tic-like behaviours in children and adolescents during COVID-19: Clinical features, assessment and biopsychosocial treatment approach.

Rapid onset functional tic-like behaviours in children and adolescents during COVID-19: Clinical features, assessment and biopsychosocial treatment approach.

Journal: Journal of paediatrics and child health

Published: August 10, 2021

Psychiatric comorbidity is common in dystonia and other movement disorders.

Psychiatric comorbidity is common in dystonia and other movement disorders.

Journal: Archives of disease in childhood

Published: April 30, 2020

Evaluation of an educational video providing key messages for doctors to counsel families following a first afebrile seizure.

Evaluation of an educational video providing key messages for doctors to counsel families following a first afebrile seizure.

Journal: Journal of paediatrics and child health

Published: December 18, 2019

Showing 1-12 of 16

Last Updated: 09/26/2026