Michelle Lorentzos
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View article: 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 Open
Aim: To report the efficacy and safety of ataluren in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD) from the phase III, 72-week, placebo-controlled period of Study 041. Materials & methods: Inclusion criteria: boys wi…
View article: Early diagnosis of Duchenne muscular dystrophy - A Treat-NMD international workshop
Early diagnosis of Duchenne muscular dystrophy - A Treat-NMD international workshop Open
View article: Mechanical Thrombectomy for Pediatric Arterial Ischemic Stroke from Acute M2 Occlusion
Mechanical Thrombectomy for Pediatric Arterial Ischemic Stroke from Acute M2 Occlusion Open
Pediatric large-vessel occlusion has a poor natural history. Recent retrospective studies have demonstrated the potential benefits, feasibility, and safety profile of mechanical thrombectomy in children. However, the role of thrombectomy i…
View article: Newborn screening for Duchenne muscular dystrophy: the perspectives of stakeholders
Newborn screening for Duchenne muscular dystrophy: the perspectives of stakeholders Open
View article: 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 Open
Opportunities for children to benefit from novel therapies have increased substantially over the past decade. Change is needed to maximise these opportunities, particularly in the paediatric trial environment. Investment and a coordinated …
View article: Newborn Screening for the Diagnosis and Treatment of Duchenne Muscular Dystrophy
Newborn Screening for the Diagnosis and Treatment of Duchenne Muscular Dystrophy Open
A pilot newborn screening (NBS) program for Duchenne muscular dystrophy (DMD) study proposes to assess the feasibility of the screening procedure, temporal course of the various steps of screening, and the public acceptability of the progr…
View article: Rapid onset functional tic‐like behaviours in children and adolescents during <scp>COVID</scp>‐19: Clinical features, assessment and biopsychosocial treatment approach
Rapid onset functional tic‐like behaviours in children and adolescents during <span>COVID</span>‐19: Clinical features, assessment and biopsychosocial treatment approach Open
Aim To report the prevalence and clinical characteristics of children with rapid onset functional tic‐like behaviours during the COVID‐19 pandemic. Methods Single centre, retrospective cohort study of children (<18 years) referred to the t…
View article: Psychiatric comorbidity is common in dystonia and other movement disorders
Psychiatric comorbidity is common in dystonia and other movement disorders Open
Objective To determine rates of psychiatric comorbidity in a clinical sample of childhood movement disorders (MDs). Design Cohort study. Setting Tertiary children’s hospital MD clinics in Sydney, Australia and London, UK. Patients Cases we…
View article: The Psychiatry of Paediatric Movement Disorders
The Psychiatry of Paediatric Movement Disorders Open
I compared the rate of psychiatric comorbidity in children with Non-tic movement disorders to children with tics and TS. In addition, this PhD explores whether children with Non-tic movement disorders have elevated rates of psychiatry comp…
View article: A review of psychiatric co-morbidity described in genetic and immune mediated movement disorders
A review of psychiatric co-morbidity described in genetic and immune mediated movement disorders Open
View article: Clinical Characteristics and Functional Motor Outcomes of Enterovirus 71 Neurological Disease in Children
Clinical Characteristics and Functional Motor Outcomes of Enterovirus 71 Neurological Disease in Children Open
Enterovirus 71 may cause serious neurological disease in young patients. The distinct clinicoradiological syndromes, predominantly within the spinal cord and brainstem, enable rapid recognition within evolving outbreaks. Long-term function…
View article: Cannabinoids for paediatric epilepsy: Weeding out the issues
Cannabinoids for paediatric epilepsy: Weeding out the issues Open
Cannabis has been considered a therapeutic herb by many civilisations across several centuries. During recent months, there has been increasing media interest and public discussion regarding a potential utility for cannabis in paediatric e…