ABSTRACT Objective Variants in SLC6A1, encoding the GABA transporter 1 (GAT‐1), cause epilepsy, autism spectrum disorder, and developmental delay via loss of GABA uptake, impaired trafficking, and ER retention. We previously found that 4‐Phenylbutyrate (PBA), an FDA‐approved drug, restores GABA uptake and reduces seizures in SLC6A1‐related disorders ...
Melissa B. DeLeeuw +5 more
wiley +1 more source
Evaluation of the Efficacy of Systemic Inflammatory Markers in Predicting Medical Treatment Failure in Patients with Tubo-Ovarian Abscess. [PDF]
Akıncı GE +6 more
europepmc +1 more source
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
Treatment Failure and Overall Survival in Patients with Sinonasal Squamous Cell Carcinoma (SNSCC): A Systematic Review and Meta-Analysis. [PDF]
Kacorzyk U +5 more
europepmc +1 more source
T1 Over Squared Proton Density Ratio to Characterize Multiple Sclerosis Lesions
ABSTRACT Objective Differentiating remyelinated from demyelinated lesions in MS remains challenging without histological confirmation. This study introduces the T1‐to‐PD2 ratio (TPR) imaging approach and evaluates its ability to characterize MS lesions alongside other quantitative MRI (qMRI) metrics. Methods Thirty individuals with MS (mean age: 47.5 ±
Sarah J. Wright +10 more
wiley +1 more source
Risk factors for treatment failure in lactational mastitis: a retrospective cohort study in China. [PDF]
Duan Z +7 more
europepmc +1 more source
Cognitive and Neuroimaging Divergence Between Juvenile and Adult FUS Amyotrophic Lateral Sclerosis
ABSTRACT Objective Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disorder characterized by progressive motor neuron degeneration. Fused in sarcoma (FUS)‐associated juvenile ALS (jALS) represents a distinct and aggressive subgroup with rapid deterioration and poor prognosis.
Alexandra V. Jürs +7 more
wiley +1 more source
Treatment failure in a dog with acute Babesia canis infection using lower-range therapeutic doses of imidocarb dipropionate. [PDF]
Schäfer I +6 more
europepmc +1 more source
Predicting Treatment Failure With Sodium-Glucose Cotransporter-2 Inhibitors in People With Type 2 Diabetes: Novel Artificial Intelligence and Machine Learning Approach. [PDF]
Kwak D +5 more
europepmc +1 more source

