Purpose: Alimentary tract duplications (ATDs) are rare congenital lesions often associated with anomalies such as spinal, urinary and GI tract malformations. The purpose of this study was to report the experience of a single center with ATDs in children, focusing on the natural history, associated malformations, and their impact on patient management.
Methods: We performed a retrospective analysis over 14 years, collecting prenatal, clinical, surgical, and follow-up data.
ACS Synth Biol
October 2024
The design of biomanufacturing platforms based on viral transduction and/or propagation poses significant challenges at the intersection between synthetic biology and process engineering. This paper introduces vitraPro, a software toolkit composed of a multiscale model and an efficient numeric technique that can be leveraged for determining genetic and process designs that optimize transduction-based biomanufacturing platforms and viral amplification processes. Viral infection and propagation for up to two viruses simultaneously can be simulated through the model, considering viruses in either the lytic or lysogenic stage, during batch, perfusion, or continuous operation.
View Article and Find Full Text PDFBackground: Percutaneous endoscopic gastrostomy is commonly used for enteral nutritional access, but gastrocutaneous fistulae (GCF) may persist after tube removal, posing clinical challenges. The use of endoscopic closure devices, including over-the-scope clips (OTSC), has shown promise in managing non-healing fistulae, although data in the pediatric population are limited.
Methods: A retrospective multicenter study analyzed pediatric patients who underwent GCF closure following gastrostomy tube removal.
Current processes for the production of recombinant adeno-associated virus (rAAV) are inadequate to meet the surging demand for rAAV-based gene therapies. This article reviews recent advances that hold the potential to address current limitations in rAAV manufacturing. A multidisciplinary perspective on technological progress in rAAV production is presented, underscoring the necessity to move beyond incremental refinements and adopt a holistic strategy to address existing challenges.
View Article and Find Full Text PDFPancreatitis, in general, is a high-morbidity condition. Genetic conditions and anatomic variants are sometimes seen, especially in children, where biliary etiologies and alcohol are less common than in adults. The decision to intervene, the combined operative-endoscopic strategy, and the timing pose unique challenges.
View Article and Find Full Text PDFIn this work dynamic models of the continuous crystallization, filtration, deliquoring, washing, and drying steps are introduced, which are developed in the open-source pharmaceutical modeling tool PharmaPy. These models enable the simulation and digital design of an integrated continuous two-stage crystallization and filtration-drying carousel system. The carousel offers an intensified process that can manufacture products with tailored properties through optimal design and control.
View Article and Find Full Text PDFBackground: Mobile technology is increasingly prevalent in healthcare, serving various purposes, including remote health monitoring and patient self-management, which could prove beneficial to early hospital discharges.
Aims: This study investigates the transitional care program experience facilitating early discharges in a pediatric setting through the use of an easy-to-use mobile medical device (TytoCare™, TytoCare Ltd., Natanya, Israel).
Surgical techniques are evolving in Pediatric Surgery, especially in the area of minimal access surgery (MAS) where indications for applications are expanding. Miniaturization of instruments, using natural orifices, single incisions, or remotely controlled robot-assisted procedures, promises to increase the benefits of MAS procedures in pediatrics. Many pediatric pathologies are rare, and specialized surgical and anesthesiologic instruments are necessary to manage them, defined as "orphan devices", for which development and dissemination on the market are slowed down or sometimes hindered by regulatory standards and limiting financial conflicts of interest.
View Article and Find Full Text PDFEosinophilic gastrointestinal diseases (EGIDs) are rare, chronic inflammatory disorders characterized by eosinophilic infiltration of the gastrointestinal tract. Symptoms and clinical presentations vary depending on the site and layer of the gastrointestinal wall infiltrated by eosinophils. Gastrointestinal obstruction is a serious, though uncommon, presentation.
View Article and Find Full Text PDFPoly(ethylene terephthalate) (PET) is a major plastic polymer utilized in the single-use and textile industries. The discovery of PET-degrading enzymes (PETases) has led to an increased interest in the biological recycling of PET in addition to mechanical recycling. IsPETase from Ideonella sakaiensis is a candidate catalyst, but little is understood about its structure-function relationships with regards to PET degradation.
View Article and Find Full Text PDFBackground: In children, laparoscopic cholecystectomy (LC) is now considered the gold standard for gallbladder (GB) removal. In the past, hemolytic disorders associated with cholelithiasis represented the most frequent conditions requiring LC; this is being overtaken by cholelithiasis and biliary conditions in overweight or ex-premature children.
Aims: This study aims to describe current indications and timing for LC in pediatric patients.
Current manufacturing processes for recombinant adeno-associated viruses (rAAVs) have less-than-desired yields and produce significant amounts of empty capsids. The increasing demand and the high cost of goods for rAAV-based gene therapies motivate development of more efficient manufacturing processes. Recently, the US Food and Drug Administration (FDA) approved the first rAAV-based gene therapy product manufactured in the baculovirus expression vector system (BEVS), a technology that demonstrated production of high titers of full capsids.
View Article and Find Full Text PDFBackground And Aims: Foreign body ingestion (FBI) in children requires early identification to prevent adverse outcomes and may necessitate endoscopic or surgical intervention. This study aims to develop a nomogram that identifies children who require urgent surgical or endoscopic intervention by using the patient's medical history and clinical parameters collected at admission.
Methods: This study is a retrospective review (01/2015-12/2020) of a multicenter case series of children admitted for FBI.
Purpose: Vessel wall imaging (VWI) with black-blood (BB) technique can demonstrate aneurysmal enhancement preluding to growth/rupture in treatment-naive cerebral aneurysms. Interestingly, recent works showed that BB enhancement may also occur in endovascularly treated aneurysms, though its meaning is controversial. Hypothesizing a flow-related mechanism of BB enhancement, we explored its relationship with incomplete occlusion status and coil packing density at DSA.
View Article and Find Full Text PDFThis Position Statement updates the different components of the therapy of obesity (lifestyle intervention, drugs, and surgery) in children and adolescents, previously reported in the consensus position statement on pediatric obesity of the Italian Society of Pediatric Endocrinology and Diabetology and the Italian Society of Pediatrics. Lifestyle intervention is the first step of treatment. In children older than 12 years, pharmacotherapy is the second step, and bariatric surgery is the third one, in selected cases.
View Article and Find Full Text PDFThe prevalence of pediatric obesity is rising rapidly worldwide, and "omic" approaches are helpful in investigating the molecular pathophysiology of obesity. This work aims to identify transcriptional differences in the subcutaneous adipose tissue (scAT) of children with overweight (OW), obesity (OB), or severe obesity (SV) compared with those of normal weight (NW). Periumbilical scAT biopsies were collected from 20 male children aged 1-12 years.
View Article and Find Full Text PDFEndoscopic procedures are performed more frequently in children due to technological advances that can be safely performed in an adequate setting with a support of a multidisciplinary team. Pediatric indications for ERCP (endoscopic retrograde cholangiopancreatography) and EUS (endoscopic ultrasound) occur mainly due to congenital malformations. In a pediatric case series, we report the application of EUS combined with duodenoscopy, eventually associated with ERCP and minimally invasive surgery, highlighting the importance of defining a tailored dedicated management pathway for each patient.
View Article and Find Full Text PDFSevere obesity remains one of the most important symptoms of Prader-Willi Syndrome (PWS), and controlling weight represents a crucial point in the therapeutical approach to the syndrome. We present an overview of different progressive patterns of growth that involve controlling weight in PWS. Mechanisms involved in the development of obesity and in preventive and therapeutic strategies to control weight gain are discussed.
View Article and Find Full Text PDFAnorectal malformations (ARMs) are rare and involve a wide spectrum of malformations. Prenatal diagnosis is often incomplete, and the diagnostic pathway is started during the newborn period to identify the type of malformation and the correct treatment. This retrospective study included patients between 8 and 18 y.
View Article and Find Full Text PDFContinuous manufacturing and closed-loop quality control are emerging technologies that are pivotal for next-generation pharmaceutical modernization. We develop a process control framework for a continuous carousel for integrated filtration-drying of crystallization slurries. The proposed control system includes model-based monitoring and control routines, such as state estimation and real-time optimization, implemented in a hierarchical, three-layer quality-by-control (QbC) framework.
View Article and Find Full Text PDFLong term follow-up of patients with esophageal atresia (EA) may be hampered by esophageal dysmotility, which affects quality of life and might lead to late complications. The endoluminal functional lumen imaging probe (EndoFlip™ Medtronic, Crospon Inc.) is an innovative diagnostic tool that assesses esophageal distensibility.
View Article and Find Full Text PDFEsophageal atresia (EA) is a congenital malformation that affects the normal esophageal development. Surgical treatment, although restoring the integrity of the alimentary tract, may lead to long-term sequelae-like developmental abnormalities and musculoskeletal deformities. We evaluated the effects of osteopathic manipulative treatment (OMT) on the recovery of the range of the right upper limb movement and on the rise of the auxological parameters.
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