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UnknownGlobal effects of non-native tree species on multiple ecosystem services.
Non-native tree (NNT) species have been transported worldwide to create or enhance services that are fundamental for human well-being, such as timber provision, erosion control or ornamental value; yet NNTs can also produce undesired effects, such as fire proneness or pollen allergenicity. Despite t...
S-EPMC6850375 | biostudies-literature
UnknownCUL3-related neurodevelopmental disorder: Clinical phenotype of 20 new individuals and identification of a potential phenotype-associated episignature.
Neurodevelopmental disorder with or without autism or seizures (NEDAUS) is a neurodevelopmental disorder characterized by global developmental delay, speech delay, seizures, autistic features, and/or behavior abnormalities. It is caused by CUL3 (Cullin-3 ubiquitin ligase) haploinsufficiency. We coll...
S-EPMC11617854 | biostudies-literature
UnknownLong-term follow-up of IPEX syndrome patients after different therapeutic strategies: An international multicenter retrospective study.

Background

Immunodysregulation polyendocrinopathy enteropathy x-linked (IPEX) syndrome is a monogenic autoimmune disease caused by FOXP3 mutations. Because it is a rare disease, the natural history and response to treatments, including allogeneic hematopoietic stem cell transplantation (HSCT...
S-EPMC6050203 | biostudies-literature
UnknownSafety of dabigatran etexilate for the secondary prevention of venous thromboembolism in children.
This open-label, single-arm, prospective cohort trial is the first phase 3 safety study to describe outcomes in children treated with dabigatran etexilate for secondary venous thromboembolism (VTE) prevention. Eligible children aged 12 to <18 years (age stratum 1), 2 to <12 years (stratum 2), and >3...
S-EPMC7019192 | biostudies-literature
UnknownCondition-Specific Growth Charts for Children With Alagille Syndrome.

Importance

Different degrees of growth delay have been reported in children with Alagille syndrome (ALGS), yet these patients are routinely evaluated using standard growth charts.

Objective

To develop condition-specific growth charts for ALGS using modern statistical approaches.

De...

S-EPMC12645329 | biostudies-literature
UnknownEvent-free survival of maralixibat-treated patients with Alagille syndrome compared to a real-world cohort from GALA.

Background and aims

Alagille syndrome (ALGS) is characterized by chronic cholestasis with associated pruritus and extrahepatic anomalies. Maralixibat, an ileal bile acid transporter inhibitor, is an approved pharmacologic therapy for cholestatic pruritus in ALGS. Since long-term placebo-cont...
S-EPMC11095900 | biostudies-literature
UnknownElevated Serum Bile Acids Predict Poor Liver Outcomes in Children With Alagille Syndrome: Results From the GALA Study Group.

Background and aim

Alagille syndrome (ALGS) is a rare disorder characterised by cholestasis and extrahepatic manifestations. Given the current era of ileal bile acid transporter (IBAT) inhibitor therapies that reduce serum bile acid (SBA) levels, we evaluated whether SBA predicts liver disea...
S-EPMC12625800 | biostudies-literature
UnknownPrognostic indicators and outcomes of hospitalised COVID-19 patients with neurological disease: An individual patient data meta-analysis.

Background

Neurological COVID-19 disease has been reported widely, but published studies often lack information on neurological outcomes and prognostic risk factors. We aimed to describe the spectrum of neurological disease in hospitalised COVID-19 patients; characterise clinical outcomes; a...
S-EPMC9162376 | biostudies-literature
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