Results 51 to 60 of about 13,060,359 (252)
Onasemnogene Abeparvovec in Patients With SMA: Interim Results of the RESTORE Registry in Japan
ABSTRACT Objective There are limited real‐world data regarding the safety and effectiveness of onasemnogene abeparvovec (OA; Zolgensma) infusion, a one‐time gene replacement therapy, for Japanese patients with spinal muscular atrophy (SMA). We aimed to improve understanding of the real‐world outcomes for OA in Japan.
Kayoko Saito +8 more
wiley +1 more source
Objective To investigate the potential predictive value of novel inflammatory markers for adverse pregnancy outcomes in pregnant women with polycystic ovary syndrome (PCOS). Methods A retrospective case‒control study was conducted.
Na Pan +6 more
doaj +1 more source
A novel variant of the SOX10 gene associated with Waardenburg syndrome type IV
Background Waardenburg syndrome (WS) is a rare genetic disorder characterized by varying degrees of sensorineural hearing loss and accumulated pigmentation in the skin, hair and iris.
Yanan Wang +3 more
doaj +1 more source
Digital Cognitive Testing in Mitochondrial Disease: Validity and Challenges for Clinical Trial Use
ABSTRACT Background Primary mitochondrial disease is a group of genetic disorders caused by pathogenic variants in nuclear or mitochondrial DNA, often resulting in progressive neurodegeneration and cognitive decline. Current management is primarily supportive, though recent research offers hope for disease‐modifying treatments in the future.
Oksana Pogoryelova +9 more
wiley +1 more source
Published FY 90-FY 93 by the Bureau of Maternal and Child Health; FY 94-FY 96 by Bureau for Children, Youth and ...
Kansas. Bureau of Maternal and Child Health
core +1 more source
Objective Reproductive‐age women with systemic autoimmune and rheumatic diseases (SARDs) have unique information needs related to their SARDs and reproductive health. We sought to understand their use of and receptivity to current and hypothetical generative artificial intelligence (AI) tools for health information‐seeking. Methods We conducted a cross‐
Mariam Arif +5 more
wiley +1 more source
Published FY 90-FY 93 by the Bureau of Maternal and Child Health; FY 94-FY 96 by Bureau for Children, Youth and ...
Kansas. Bureau of Maternal and Child Health
core +1 more source
A glial cell line‐derived neurotrophic factor (GDNF)‐enriched small extracellular vesicle‐loaded reactive oxygen species (ROS)‐scavenging hydrogel integrates sustained neurotrophic delivery with local modulation of oxidative and inflammatory stress after spinal cord injury. The composite hydrogel improves local vesicle retention, protects neural tissue,
Xingyu Zhu +13 more
wiley +1 more source
This study identified Danofloxacin as a novel supporter of the maintenance of mouse embryonic stem cell (ESC) pluripotency. It functions partially by inhibiting HDAC1, which increases specific histone acetylation marks and activates key genes like Tert and Prdm10.
Yan Zhang +8 more
wiley +1 more source
ABSTRACT Autism Spectrum Disorder (ASD) is associated with gut microbiota dysbiosis, yet the contribution of specific microbial metabolic pathways remains unclear. Reanalysis of a public ASD cohort revealed reduced functional potential in microbial tryptophan oxidative metabolism.
Rui Guo +14 more
wiley +1 more source

