Annals of Clinical and Translational Neurology, EarlyView.ABSTRACT Objective Onasemnogene abeparvovec (OA) is an AAV9‐based gene therapy for spinal muscular atrophy type I (SMA I). Real‐world outcomes show increased response variability compared to clinical trials, and follow‐up data beyond 12–18 months are limited.
Marika Pane +43 more
wiley +1 more source
Parental Low Level of Education and Single-Parent Families as Predictors of Poor Control of Type 1 Diabetes in Children Followed in French Guiana. [PDF]
Int J Environ Res Public HealthSamou-Fantcho CB +3 more
europepmc +1 more source
Single-Parent Expression of Anti-sense RNA Contributes to Transcriptome Complementation in Maize Hybrid. [PDF]
Front Plant Sci, 2020 Zhang X, Qi Y.
europepmc +1 more source
Cracking the Code: Genotype–Phenotype Correlation Models in Sarcoglycanopathies
Annals of Clinical and Translational Neurology, EarlyView.ABSTRACT Objective Sarcoglycanopathies are among the most severe limb‐girdle muscular dystrophies (LGMD), though milder presentations have been described. These diseases are primarily caused by missense variants, but the limited predictability of their effect on protein maturation, complex formation, and transport has hindered reliable genotype ...
Leonela Luce +72 more
wiley +1 more source
The influence of left-behind experiences on depression in single-parent adolescents: the mediating role of family satisfaction and the moderating effects of exercise frequency. [PDF]
Front PediatrCen WC +4 more
europepmc +1 more source
A Depolarizing Leak in Sodium Bicarbonate Cotransporter NBCe1 Causes Brain Edema
Annals of Clinical and Translational Neurology, EarlyView.ABSTRACT Objectives SLC4A4 encodes electrogenic sodium bicarbonate cotransporter NBCe1, prominently expressed in kidney and brain. Recessive loss‐of‐function variants in SLC4A4 cause proximal renal tubular acidosis, no brain edema. In the brain, NBCe1 is expressed by astrocytes, where it regulates pH and mediates astrocyte volume changes.
Quinty Bisseling +16 more
wiley +1 more source
Correction: Effect of single-parent versus dual-parent households on dietary intake and growth among under-five children in Rwanda: an analysis using directed acyclic graph. [PDF]
BMC NutrUkwishaka J +4 more
europepmc +1 more source
Single-parent status and smoke-free home rules among daily smokers. [PDF]
Can J Public Health, 2020 Montreuil A, Wellman RJ, O'Loughlin JL.
europepmc +1 more source
Multiple Sclerosis Relapse Activity After Ozanimod Discontinuation in DAYBREAK Trial Participants
Annals of Clinical and Translational Neurology, EarlyView.Multiple Sclerosis Relapse Activity After Ozanimod Discontinuation in DAYBREAK Trial Participants. ABSTRACT Objective Return of disease activity is expected when patients discontinue disease‐modifying therapy (DMT) for multiple sclerosis (MS). Some MS DMTs are associated with higher‐than‐expected disease activity (rebound) after discontinuation.
Ralf Gold +12 more
wiley +1 more source
Effect of single-parent versus dual-parent households on dietary intake and growth among under-five children in Rwanda: an analysis using directed acyclic graph. [PDF]
BMC NutrUkwishaka J +4 more
europepmc +1 more source