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International Consensus Recommendations for the Assessment and Management of Individuals With CDKL5 Deficiency DisorderCDKL5 Deficiency Disorder (CDD) is a rare, X-linked dominant condition that causes a developmental and epileptic encephalopathy (DEE). The incidence is between ~ 1:40,000 and 1:60,000 live births. Pathogenic variants in CDKL5 lead to seizures from infancy and severe neurodevelopmental delay.
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ATAGI 2022 Annual Statement on ImmunisationChristopher Blyth MBBS (Hons) DCH FRACP FRCPA PhD Centre Head, Wesfarmers Centre of Vaccines and Infectious Diseases; Co-Head, Infectious Diseases
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Short Message Service Reminder Nudge for Parents and Influenza Vaccination Uptake in Children and Adolescents with Special Risk Medical Conditions: The Flutext-4U Randomized Clinical TrialChildren with chronic medical conditions are at increased risk of severe influenza. Uptake of influenza vaccination in children and adolescents with these identified special risk medical conditions is suboptimal.
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Childhood-onset type 1 diabetes in Western Australia: An update on incidence and temporal trends from 2001 to 2022To determine the incidence and incidence trends over 2001-2022 of childhood-onset type 1 diabetes (T1D) in Western Australia and assess the impact of the COVID-19 pandemic.
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No association between in utero exposure to emissions from a coalmine fire and post-natal lung functionStudies linking early life exposure to air pollution and subsequent impaired lung health have focused on chronic, low-level exposures in urban settings. We aimed to determine whether in utero exposure to an acute, high-intensity air pollution episode impaired lung function 7-years later.
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Blinatumomab Added to Chemotherapy in Infant Lymphoblastic LeukemiaKMT2A-rearranged acute lymphoblastic leukemia (ALL) in infants is an aggressive disease with 3-year event-free survival below 40%. Most relapses occur during treatment, with two thirds occurring within 1 year and 90% within 2 years after diagnosis. Outcomes have not improved in recent decades despite intensification of chemotherapy.
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No Health without Mental Health: Taking Action to Heal a World in Distress—With People, Places, and Planet ‘in Mind’The unprecedented global rise in mental anguish is closely linked with the erosion of our social fabric, economic and political systems, and to our natural environments. We are facing multiple new large-scale threats to health, safety, and security, with a growing lack of trust in others and in authorities.
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Acute Lymphoblastic Leukemia in Infants: A Distinctive, High-Risk Subtype of Childhood Acute Lymphoblastic LeukemiaAcute lymphoblastic leukemia (ALL) in infants younger than 1 year of age is an aggressive, high-risk subtype of childhood ALL. Infant ALL with KMT2A-r is characteristically poorly responsive to chemotherapy and hematopoietic stem cell transplantation. New strategies, such as molecularly targeted therapies and immunotherapies, are in development and show promise in preclinical models and early phase studies.
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Parents' COVID-19 vaccine intentions for children under 5 years: Brief reflections from a qualitative studyAustralian authorities made COVID-19 vaccines available for children aged under 5 years old with serious comorbidities in August 2022. There is presently no universal programme for young children, but crucial to any rollout's success is whether parents are motivated and able to vaccinate. By examining parents' vaccine intentions, this study aims to inform current and future COVID-19 vaccine roll-outs for children aged under 5 years.
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Survival Outcomes of Children with Relapsed or Refractory Myeloid Leukemia Associated with Down syndromeChildren with Down syndrome (DS) are at a significantly higher risk of developing acute myeloid leukemia, also termed myeloid leukemia associated with DS (ML-DS). In contrast to the highly favorable prognosis of primary ML-DS, the limited data that are available for children who relapse or who have refractory ML-DS (r/r ML-DS) suggest a dismal prognosis. There are few clinical trials and no standardized treatment approach for this population.