NYU Langone Health

Efficacy and safety of fenfluramine in Dravet syndrome: The impact of patient clinical characteristics

Abstract Objective To assess the efficacy and safety of fenfluramine in patients with Dravet syndrome (DS) stratified by age, number of previously attempted antiseizure medications (ASMs), and SCN1A pathogenic variant status. Methods In this post hoc analysis, data from three ran...

SPINK1-COL18A1 crosstalk shapes epigenome and drives cancer stemness in pancreatic ductal adenocarcinoma

Pancreatic ductal adenocarcinoma (PDAC) is a devastating cancer with an increasing incidence and extremely dismal prognosis. Discovery and mechanistic understanding of the genetic and epigenetic drivers of PDAC is therefore of critical importance. Here, we uncover serine protease...

The Dutch Prolactinoma Cohort Studies: Cross-sectional Analysis of Females Focusing on the Patient's Perspective

Abstract Purpose To assess the results of patient-reported outcome measures (PROMs) in a national cohort of females with a prolactinoma by disease activity. Methods Females with prolactinoma included in the PRolaCT or ProlaC study were assessed cross-sectionally, comparing patien...

Failure Modes of Total Wrist Arthroplasty: Cross-Sectional Analysis of FDA MAUDE Adverse Event Reports (2015-2025)

Background: Total wrist arthroplasty (TWA) is a motion-preserving alternative to wrist fusion for end-stage wrist arthritis, but concerns regarding long-term durability continue to limit broader use. Reported 10-year survivorship ranges from 70% to 80%, although real-world failur...

Redefining Progression in a Rapidly Evolving Therapeutic Landscape

This Viewpoint the regulatory problem of how to design clean trials that regulators seek as the field of oncology becomes more therapeutically sophisticated.

Acquired resistance to the RAS(ON) multi-selective inhibitor daraxonrasib guides rational combination therapy strategies in pancreatic cancer

Abstract Daraxonrasib is an orally bioavailable RAS(ON) multi-selective tri-complex inhibitor of the oncogenic mutant and wild-type variants of N, H and KRAS. We previously reported encouraging efficacy in a phase 1/2 clinical trial evaluating daraxonrasib monotherapy at clinical...

Prenatal therapies to improve outcomes in gastroschisis: a systematic scoping review protocol

INTRODUCTION: Gastroschisis is a congenital birth defect with a rising incidence. The herniation of intestines into the amniotic cavity leads to prenatal bowel injury, which contributes to significant postnatal morbidity and mortality. Current management focuses on postnatal inte...

Management of high-risk (B3) breast lesions in symptomatic practice: evidence review and practical recommendations for multidisciplinary care

Borderline breast lesions (B3 lesions, also termed lesions of uncertain malignant potential or high-risk lesions) represent a heterogeneous group of entities associated with variable risks of malignancy. While the management of screen-detected B3 lesions has become increasingly s...

A Retrospective Clinical Study of 44 Cases of Parapharyngeal Space Tumors

副咽頭間隙腫瘍はまれな疾患であり，治療方針の決定に苦慮することが多い。本検討では，手術適応の決定に関連する臨床因子を探索することを目的として，当院で診療した44例を後方視的に解析した。初回治療方針により手術群16例，経過観察群28例に分類し，性別，年齢，症状，腫瘍体積，腫瘍位置，FNAC施...

Safety and Tolerability of Chronic Carvedilol in Pediatric Dilated Cardiomyopathy: A Report from the Pediatric Cardiomyopathy Registry

Long-term studies on safety of beta-blockers in pediatric heart failure (HF) are scant. We describe the safety profile, dose ranges, and tolerability of carvedilol in pediatric dilated cardiomyopathy (DCM). The Pediatric Cardiomyopathy Registry (PCMR) was used to identify patient...

“Orthoplastic” Collaboration in fracture-related infection management reflects case complexity and is associated with achievable infection control

Background: The purpose of this study was to characterize fracture-related infection (FRI) cases requiring plastic surgery involvement and to evaluate infection-related outcomes in the setting of “orthoplastic collaboration.” Methods: A retrospective review was conducted of all p...

Spatially resolved tissue architecture and computational pathology in pancreatic cancer

Pancreatic ductal adenocarcinoma (PDAC) is a complex disease characterized by high levels of cellular heterogeneity and pronounced microenvironmental remodelling. Dynamic changes during its initiation and progression contribute to resistance to conventional therapies. Building up...

RAS inhibition limits oncogenic signaling and tumorigenesis in neuroblastoma regardless of RAS mutational status

Abstract Neuroblastoma (NBL) is a rare and aggressive pediatric malignancy with a 5-year survival rate of 40–50% for patients diagnosed with high-risk disease. NBLs arise in the sympathetic nervous system and causes 11% of all pediatric cancer deaths. MYCN amplification or overex...