Results 111 to 120 of about 823,547 (269)

Plasma EV Proteomics Identifies ECM Remodeling and Inflammatory Proteins LUM and C7 as Candidate Biomarkers in FSHD

open access: yesAnnals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Facioscapulohumeral muscular dystrophy (FSHD) is one of the most debilitating and common muscular dystrophies. Despite its severity, no approved therapy exists for FSHD patients. However, several therapeutic candidates are currently under development, and some have recently entered clinical trials, marking the need for reliable ...
Mustafa Bilal Bayazit   +11 more
wiley   +1 more source

Onasemnogene Abeparvovec in Patients With SMA: Interim Results of the RESTORE Registry in Japan

open access: yesAnnals of Clinical and Translational Neurology, EarlyView.
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

Stage‐Dependent β‐Synuclein Links MRI and Cognitive Decline in Alzheimer's Disease

open access: yesAnnals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Synaptic degeneration drives cognitive decline in Alzheimer's disease (AD), but synaptic biomarkers are scarce. Brain‐enriched β‐synuclein emerged as a synaptic damage marker. We investigated its diagnostic, prognostic, and structural correlates across the AD continuum.
Ulaş Ay   +15 more
wiley   +1 more source

Augmenting and Assaying Nav1.1 Protein Quantity for Dravet Syndrome Therapy

open access: yesAnnals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Dravet Syndrome (DS) is a developmental and epileptic encephalopathy predominantly caused by heterozygous loss‐of‐function variants in SCN1A, which encodes Nav1.1. Conserved upstream open reading frames (uORFs) in SCN1A were validated to regulate translation in reporter assays, demonstrating the therapeutic viability of increasing Nav1.1 from ...
Aiswarya Saravanan   +7 more
wiley   +1 more source

RFAmide-Related Peptide: Another Sexy Peptide? [PDF]

open access: yesEndocrinology, 2008
Ebling, Francis J P   +1 more
openaire   +3 more sources

Combining Three Peripheral Blood Biomarkers to Stratify Rheumatoid Arthritis–Associated Interstitial Lung Disease Risk

open access: yesArthritis Care &Research, EarlyView.
Objective The purpose was to evaluate a biomarker score consisting of MUC5B rs35705950 promoter variant, plasma matrix metalloproteinase‐7 (MMP‐7), and serum anti–malondialdehyde‐acetaldehyde (anti‐MAA) antibody for rheumatoid arthritis (RA)–associated interstitial lung disease (ILD) risk stratification.
Kelsey Coziahr   +16 more
wiley   +1 more source

Cementum Attachment Protein-Derived Peptides Modulate Brushite and Calcium Oxalate Crystallization In Vitro. [PDF]

open access: yesJ Pept Sci
Mimila-Cortes A   +9 more
europepmc   +1 more source

Use of a Molecular Signature Response Classifier to Inform Treatment Selection Improves Clinical Disease Activity Among Patients with Rheumatoid Arthritis Initiating a Biologic or Targeted Synthetic Disease‐Modifying Antirheumatic Drug

open access: yesArthritis Care &Research, EarlyView.
Objective We assessed the effectiveness of PrismRA to improve clinical outcomes among patients with rheumatoid arthritis (RA) initiating treatment with a biologic or targeted synthetic disease‐modifying antirheumatic drug (b/tsDMARD). Methods PrismRA incorporated 19 gene expression features and four clinical features to assess a patient's likelihood of
Fenglong Xie   +3 more
wiley   +1 more source

The Dual Role of Natural Peptides in Cancer Therapy: Anticancer and Immunomodulatory Perspectives. [PDF]

open access: yesOncol Res
Tsaturyan A   +7 more
europepmc   +1 more source

Evaluating a Pragmatic Strength Alternative for Frailty Measurement and Assessing Its Predictive Capacity Against Established Frailty Instruments in Rheumatoid Arthritis

open access: yesArthritis Care &Research, EarlyView.
Objective Frailty occurs prematurely in rheumatoid arthritis (RA) and is associated with poor health outcomes. We compared the performance of four frailty instruments, including a pragmatic alternative measure using chair sit‐to‐stand (STS), and evaluated their abilities to predict poor health outcomes.
Kylie E. Riggles   +14 more
wiley   +1 more source

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