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C1 CAGE detects transcription start sites and enhancer activity at single-cell resolution.We introduce C1 CAGE, a method for the detection of transcript 5'-ends with an original sample multiplexing strategy in the C1TM microfluidic system
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Interleukin-1 is associated with inflammation and structural lung disease in young children with cystic fibrosisOur data associates IL-1α with early structural lung damage in CF and suggests this pathway as a novel anti-inflammatory target
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A novel, palatable paediatric oral formulation of midazolam: Pharmacokinetics, tolerability, efficacy and safetyWe conclude that the novel chocolate-based formulation of midazolam provides improved tolerability while remaining efficacious
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Predicting respiratory hospital admissions in young people with cerebral palsyMost risk factors for respiratory hospital admissions in young people with cerebral palsy are potentially modifiable
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Expansion of the Human Phenotype Ontology (HPO) knowledge base and resourcesThe Human Phenotype Ontology is a standardized vocabulary of phenotypic abnormalities used by researchers, clinicians, informaticians and electronic health record systems
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Severity Assessment in CDKL5 Deficiency DisorderA severity assessment was rapidly developed with input from multiple stakeholders. Refinement through ongoing validation is required for future clinical trials.
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Most clinical anti-EGFR antibodies do not neutralize both wtEGFR and EGFRvIII activation in gliomaWe discovered a previously unknown major resistance mechanism in glioma in that most EGFR domain III-targeting antibodies do not neutralize EGFRvIII
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International experience in the development of patient-derived xenograft models of diffuse intrinsic pontine gliomaThis multi-center study provides valuable information on the success rate of establishing patient-derived pre-clinical models of diffuse intrinsic pontine glioma
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Planetary Health: From the Wellspring of Holistic Medicine to Personal and Public Health ImperativeHere, we explore the history of planetary health and describe its relevance to contemporary healthcare
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Facilitators and Barriers of Participation in “Uptime” Activities in Girls and Women With Rett Syndrome: Perspectives From Parents and ProfessionalsThis study explored facilitators and barriers to "uptime" (non-sedentary) activities in Danish girls and women with Rett syndrome