Novartis (Switzerland)

A Versatile DNA‐Encoded Library Platform for the Discovery of Highly Cooperative Chemical Inducers of Proximity

ABSTRACT Chemical‐induced proximity, particularly via molecular glues, represents a rapidly advancing therapeutic paradigm. Expanding the scope of proximity‐based therapeutics requires generalizable discovery platforms that can be easily tailored to diverse presenter proteins. He...

Real-World Treatment Patterns in Patients with Spinal Muscular Atrophy Receiving Multiple Disease-Modifying Therapies

Nusinersen (approved in 2016), onasemnogene abeparvovec (intravenous infusion [OAV], 2019; intrathecal injection, 2025), and risdiplam (2020) are disease-modifying therapies (DMTs) available in the US for spinal muscular atrophy (SMA). Real-world evidence on treatment patterns am...

Synthesis, Evaluation, and Context for Applications of SF5-Containing [1.1.1]Bicyclopentane and [ 2 ]Staffane “Hybrid Bioisosteres”

Abstract Bioisosteric replacements are conventionally applied as single-variable changes to a parent molecule during drug discovery campaigns. Herein, we evaluate hybrid bioisosteric replacements as strategic combinations of multiple substitutions that counterbalance the physicoc...

Comparing YAPWW1 and TAZWW: Similar Binding Sites but Different Stability and Conformational Dynamics

The two paralogs YAP and TAZ act through TEAD transcription factors and bind PPxY motif proteins in the Hippo pathway via WW domains. YAP has up to two WW domains, whereas TAZ has one. Because YAPWW1 and TAZWW are the most similar in sequence, they can be considered as correspond...

Adverse events, quality of life, and work impairment in patients with chronic myeloid leukemia (CML)—a survey of first- and second-line tyrosine kinase inhibitor (TKI) intolerance in the US

Tyrosine kinase inhibitors (TKIs) improve survival for people with chronic myeloid leukemia (CML) but may cause adverse events (AEs) that impair quality of life (QoL). Adults with CML receiving first- or second-line TKI therapy in the United States (US) completed validated questi...

Effects of hyperoxia on pulmonary inflammation and organ injury in a human in vivo model (HIPI): a randomised controlled trial

Introduction It is uncertain whether hyperoxia contributes to adverse intensive care unit outcomes. Potential harm is likely mediated by oxidative stress driving pulmonary and systemic inflammation. Methods We conducted a randomised, double-blind, placebo-controlled trial to test...

Multiple-use prediction and calibration for all future values: Exact simultaneous tolerance bands for multiple and polynomial regressions

Multiple-use prediction and calibration for all future values play an important role in many areas, including health and medical research. Simultaneous tolerance bands (STBs) can be employed for these purposes. This article studies the construction of exact STBs for multiple regr...

A community-validated tool for clinical outcome calculation in multiple sclerosis: Consensus development and scenario-specific recommendations

Background: Reproducibility and comparability of disability outcomes remain major challenges in multiple sclerosis (MS) research. Definitions of disability progression vary widely across studies, and calculation criteria are often insufficiently documented to enable replication....

The Hsd17b13P260S patient variant knock-in male mouse model provides translational insights into HSD17B13 biology

Background Metabolic dysfunction-associated steatotic liver disease (MASLD) is a prevalent liver disease that can progress to metabolic dysfunction-associated steatohepatitis (MASH), a condition marked by inflammation and fibrosis that increases the risk of cirrhosis, liver failu...

Serum Glial Fibrillary Acidic Protein Dynamics, Disease Progression, and Therapy Response in Multiple Sclerosis

Importance: Capturing individual multiple sclerosis (MS) progression is difficult; few studies have evaluated glial fibrillary acidic protein (GFAP) in large longitudinal cohorts with independent validation. Objective: To investigate whether serum GFAP levels and treatment-relate...