Characteristics of Inherited Metabolic Disorders Following Kidney Transplantation: A 13-Year Observational Study.

Inherited metabolic disorders (IMDs), primarily cystinosis, Fabry disease, and methylmalonic acidemia (MMA), are genetic conditions that typically result in multi-organ disease manifestations. Kidney function progressively deteriorates in many cases, with patients eventually reaching end-stage kidney disease (ESKD) and requiring renal replacement therapy. Kidney transplantation has been deemed the optimal renal replacement therapy option to achieve long-term survival in patients with IMD.

Vanadium complexes as potential metal-based antimicrobial drugs.

Radical increase of antibiotic resistance among microbes has become a serious problem for clinics all over the world that has led to the need for search of novel types of antimicrobial drugs. Each year, researchers synthesize a multitude of compounds in pursuit of identifying potential chemotherapeutic agents through diverse methodological evaluations. Among the vast array of biologically significant compounds, coordination compounds exhibit a broad range of activities within biological systems.

Retrospective analysis of arginase 1 deficiency progression in adults over 5 years at a single metabolic centre.

Background: The clinical presentation of ARG1-D is characterised by elevated arginine levels leading to neurological and mobility impairments. Information about long-term outcomes in adults is lacking, which prompted us to undertake a retrospective observational study.

Methods: We extracted ARG1-D patient data spanning a 5-year period from electronic health records.

Horizontal gene transfer of the Mer operon is associated with large effects on the transcriptome and increased tolerance to mercury in nitrogen-fixing bacteria.

Background: Mercury (Hg) is highly toxic and has the potential to cause severe health problems for humans and foraging animals when transported into edible plant parts. Soil rhizobia that form symbiosis with legumes may possess mechanisms to prevent heavy metal translocation from roots to shoots in plants by exporting metals from nodules or compartmentalizing metal ions inside nodules. Horizontal gene transfer has potential to confer immediate de novo adaptations to stress.

Unveiling the potential of ursolic acid modified hyaluronate nanoparticles for combination drug therapy in triple negative breast cancer.

Triple negative breast cancer (TNBC) represents the most aggressive and heterogenous disease, and combination therapy holds promising potential. Here, an enzyme-responsive polymeric prodrug with self-assembly properties was synthesized for targeted co-delivery of paclitaxel (PTX) and ursolic acid (UA). Hyaluronic acid (HA) was conjugated with UA, yielding an amphiphilic prodrug with 13.

Systemic Sclerosis in Males-Deciphering the Enigma of Erasmus Syndrome.

Background: Systemic sclerosis is an autoimmune connective tissue disease characterized by fibrosis in skin and internal organs. Chronic exposure to silica may not only lead to silicosis of lungs but also systemic sclerosis. Systemic sclerosis is relatively commoner in females; however, occupational exposure to silica in males makes them vulnerable to silica--associated systemic sclerosis (Erasmus syndrome).

HYPofractionated Adjuvant RadioTherapy in 1 versus 2 weeks in high-risk patients with breast cancer (HYPART): a non-inferiority, open-label, phase III randomised trial.

Background: Breast cancer is the most common cancer in women. Radiotherapy is an important part of breast cancer treatment after surgery. Breast cancer radiotherapy is usually delivered in 3-5 weeks.

The incidence of movement disorder increases with age and contrasts with subtle and limited neuroimaging abnormalities in argininosuccinic aciduria.

Argininosuccinate lyase (ASL) is integral to the urea cycle detoxifying neurotoxic ammonia and the nitric oxide (NO) biosynthesis cycle. Inherited ASL deficiency causes argininosuccinic aciduria (ASA), a rare disease with hyperammonemia and NO deficiency. Patients present with developmental delay, epilepsy and movement disorder, associated with NO-mediated downregulation of central catecholamine biosynthesis.

Delineating the Boundaries of Superficial Lymphangitis: A Retrospective Study of 11 Cases with a Review of Literature.

Background: Lymphangitis is an inflammation of lymphatic channels caused by infectious or non-infectious agents, presenting with characteristic linear erythematous streaks draining toward regional lymph nodes.

Objective: To describe the clinical characteristics and etiological factors involved in acute superficial lymphangitis in a retrospective descriptive study. Materials and Methods: Records of patients were analyzed retrospectively who presented with linear erythematous streaks, diagnosed as superficial lymphangitis, in the outpatient department of dermatology during the last 5 years (January 2018-December 2022) in a tertiary care hospital.

Long-term administration of intravenous Trappsol® Cyclo™ (HP-β-CD) results in clinical benefits and stabilization or slowing of disease progression in patients with Niemann-Pick disease type C1: Results of an international 48-week Phase I/II trial.

Background: Niemann-Pick disease type C (NPC) is a rare, fatal, pan-ethnic, autosomal recessive lysosomal storage disease characterized by progressive major organ failure and neurodegeneration. Preclinical studies confirmed a critical role of systemically administered hydroxypropyl-β-cyclodextrin (HP-β-CD; Trappsol Cyclo™) in cholesterol metabolism and homeostasis in peripheral tissues of the body, including the liver, and in the central nervous system (CNS). Herein, the pharmacokinetics (PK), safety, and efficacy of HP-β-CD, and biomarkers of NPC were assessed in pediatric and adult patients with NPC1.

A Comparison of Antimicrobial Efficacy of Silver-based Preventive Restorations (Silver Nitrate, Silver Diamine Fluoride, and Silver Nanoparticles) against Monospecies Biofilm Model.

Aim: The ability of the () to form biofilms is not only crucial in the initiation of early childhood caries (ECC) but is also a challenge to its treatment. The current management protocols focus on remineralization and use of antimicrobial formulations which penetrate biofilms, control their formation, and decrease the incidence of caries in children. The paradigm shift toward preventive protocols and increasing antibiotic resistance rekindled the use of silver as a promising antibacterial agent.

Recent advances in lipid-based long-acting injectable depot formulations.

Long-acting injectable (LAIs) delivery systems sustain the drug therapeutic action in the body, resulting in reduced dosage regimen, toxicity, and improved patient compliance. Lipid-based depots are biocompatible, provide extended drug release, and improve drug stability, making them suitable for systemic and localized treatment of various chronic ailments, including psychosis, diabetes, hormonal disorders, arthritis, ocular diseases, and cancer. These depots include oil solutions, suspensions, oleogels, liquid crystalline systems, liposomes, solid lipid nanoparticles, nanostructured lipid carriers, phospholipid phase separation gel, vesicular phospholipid gel etc.

Randomized Controlled Trial Comparing Ferrous Sulfate and Iron Sucrose in Iron Deficiency Anemia in Pregnancy.

Introduction:  Anemia among pregnant women is one of the major health concerns for healthcare workers. The management becomes a concern in the pregnancy where the question arises of which is better the intravenous iron sucrose or the oral ferrous sulfate tablets. To answer this, a randomized control trial comparing both the treatment options in a tertiary care government hospital was set up in the hilly terrains of India.

Improving Delirium Assessment in Palliative Homecare - A Quality Improvement Project at CanSupport.

Objectives: Delirium increases distress in patients and caregivers and often leads to hospitalisation and increased health-care costs. It is early diagnosis and management improves the quality of life (QoL) of advanced cancer patients as well as their families. This quality improvement (QI) project aimed at increasing delirium assessment in poor performance advanced cancer patients receiving palliative homecare.

Efficacy and Safety of N-Acetyl-l-Leucine in Children and Adults With GM2 Gangliosidoses.

Background And Objectives: GM2 gangliosidoses (Tay-Sachs and Sandhoff diseases) are rare, autosomal recessive, neurodegenerative diseases with no available symptomatic or disease-modifying treatments. This clinical trial investigated N-acetyl-l-leucine (NALL), an orally administered, modified amino acid in pediatric (≥6 years) and adult patients with GM2 gangliosidoses.

Methods: In this phase IIb, multinational, open-label, rater-blinded study (IB1001-202), male and female patients aged ≥6 years with a genetically confirmed diagnosis of GM2 gangliosidoses received orally administered NALL for a 6-week treatment period (4 g/d in patients ≥13 years, weight-tiered doses for patients 6-12 years), followed by a 6-week posttreatment washout period.

Self-nanoemulsifying formulation for oral delivery of sildenafil: effect on physicochemical attributes and in vivo pharmacokinetics.

Sildenafil (SLD) is employed for the management of erectile dysfunction and pulmonary arterial hypertension. It exhibits meagre water solubility and is available in the form of citrate salt hydrate to improve the solubility. However, it still exhibits moderate solubility, high first-pass metabolism, resulting in very less oral bioavailability.

Duckweed: a potential phytosensor for heavy metals.

Globally, heavy metal (HM) contamination is one of the primary causes of environmental pollution leading to decreased quality of life for those affected. In particular, HM contamination in groundwater poses a serious risk to human health and the potential for destabilization of aquatic ecosystems. At present, strategies to remove HM contamination from wastewater are inefficient, costly, laborious, and often the removal poses as much risk to the environment as the initial contamination.

Enhanced stability and oral bioavailability of erlotinib by solid self nano emulsifying drug delivery systems.

The present investigation demonstrates the preparation of solid self nanoemulsfying drug delivery system (sSNEDDS) to enhance stability and bioavailability of Erlotinib (ERL) via the oral route. Capmul®MCM EP (CPM EP, oil), Cremophor® RH 40 (CMR RH 40, surfactant), and LBF CS (LBF CS, cosurfactant) were chosen as chief components for preparing Liquids SNEDDS (L-ERL-SNEDDS) based on solubility and emulsion forming ability. Pseudo ternary phase diagram and constrained mixture designs were applied to identify the self-emulsifying area and it was found that CPM EP, CMR RH 40, and LBF CS in the ratio of 59:11:30 showed optimized particle size (110.