Stimate (desmopressin nasal spray) / CSL Behring - LARVOL DELTA

Single-nucleus RNA sequencing reveals cell types, genes, and regulatory factors influencing melanogenesis in the breast muscle of Xuefeng black-bone chicken. (PubMed, Poult Sci) - "Our findings revealed that STIMATE, LRRC7, ENSGALG00000049990, and GLDC play pivotal regulatory roles in melanin deposition within the breast muscle...This facilitated the identification of candidate genes, regulatory factors, and cellular signals potentially influencing melanin deposition and melanocyte function. Overall, our study provides crucial insights into the molecular basis of melanin deposition in chicken breast muscle, laying the groundwork for future breeding programs aimed at enhancing black-bone chicken cultivation."

Journal • CYP1B1 • GCG • IGF1 • JAG1 • KLF5 • NOTCH1 • PTPRK • ROR2

Cortical thinning and Cognition in Cerebral Small Vessel Disease (AAIC 2024) - "H igher ARTS score was associated with global cortical th inning ( e stimate= -0.14; SE=0.02, p 0.017 )... In vivo MRI -derived measure s for cerebral a rteriolosclerosis are associated with global and lobar cortical thinni ng . The association between ARTS and cognition is partially mediated by cortical thinning . Findings suggest that c erebral small vessel disease may contribute to neurodegeneration and, both directly and indirectly , to subsequent cognitive impairment."

Alzheimer's Disease • CNS Disorders • Cognitive Disorders

LIPID PROFILE AND HYPERCHOLESTEROLAEMIA OF ADOLESCENT GIRLS WITH ANOREXIA NERVOSA (ISGE 2024) - "Context - Objective : The aim of this study is to stimate the frequency and role of hypercholesterolaemia in female adolescents with Anorexia Nervosa (AN)...Nutritional recovery of AN adolescents leads to normalization of the LP profile. A multidisciplinary approach of these girls is mandatory."

Anorexia • Dyslipidemia

Prognostic factors and outcomes in neoadjuvant treatment in osteosarcoma: A single national center experience in Peru (Sarcoma-RC 2024) - "Survival analysis was performed using Kaplan Meier Stimate, also we made bivarial analysis using mortality as event of interest versus other characteristics of the patient, chemotherapy and surgery, a value of p<0.05 was considered as significant...Survival analysis was performed using Kaplan Meier Stimate, also we made bivarial analysis using mortality as event of interest versus other characteristics of the patient, chemotherapy and surgery, a value of p<0.05 was considered as significant... This is the first study describing the outcomes of neoadjuvant treatment with MAP regimen non-metastatic OS in Peru. We found that patients with joint involvement had a worse prognosis in terms of OS. Mortality increases after 20 months of follow-up."

Biomarker • Clinical • Oncology • Osteosarcoma • Sarcoma • Solid Tumor

Insights Into the Ras-MAPK Effect on Neurodevelopment: Mapping the Neuropsychiatric and Brain Phenotypes in Children With Noonan Syndrome (AACAP 2023) - "The aim was to e stimate the effect of gain-of-function mutations in the PTPN11 gene causing NS and loss-of-function mutations in the NF-1 gene on brain structure, white matter, and neurodevelopment...Since NF-1 and NS genetic mutations activate the Ras-MAPK signaling pathway, our findings provide preliminary evidence demonstrating a general effect of this activation on the brain and behavior. Divergent effects of NF-1 and NS on FA values emerge at the track level, indicating specific effects of the PTPN11 pathogenic variant."

Clinical • ADHD (Impulsive Aggression) • Attention Deficit Hyperactivity Disorder • Fibrosis • Genetic Disorders • Neurofibromatosis • Psychiatry • Solid Tumor • NF1 • PTPN11

Cross-species analysis of differential transcript usage in humans and chickens with fatty liver disease. (PubMed, Vet World) - "We identified seven genes - ALG5, BRD7, DIABLO, RSU1, SFXN5, STIMATE, TJP3, and VDAC2 - and their corresponding transcript isoforms as potential candidates (false discovery rate ≤0.05)...This study successfully identified novel human-chicken DTU genes in fatty liver disease. Further research is encouraged to verify the functions and regulations of these transcript isoforms as potential diagnostic markers for fatty liver disease in humans and chickens."

Journal • Hepatology • ALG5

Exosomal STIMATE derived from type II alveolar epithelial cells controls metabolic reprogramming of tissue-resident alveolar macrophages. (PubMed, Theranostics) - "This involves calcineurin (CaN)-PGC-1α pathway mediated mitochondrial biogenesis and mtDNA coding. In a bleomycin-induced mouse fibrosis model, supplementation with inhaled STIMATE ADEs lessened early acute injury, prevented advanced fibrosis, alleviated ventilatory impairment and reduced mortality."

Journal • Acute Respiratory Distress Syndrome • Fibrosis • Immunology • Metabolic Disorders • Pulmonary Disease • Respiratory Diseases • Transplantation

Efficacy, safety and consumption of plasma-derived von Willebrand factor (VWF)/Factor VIII (FVIII) concentrate with 2.4:1 VWF:FVIII ratio for the treatment of von Willebrand Disease: a systematic review (#74) (GTH 2023) - "Plasma-derived human coagulation FVIII/human VWF (pdVWF/FVIII; Voncento®/Biostate®) is indicated in all age groups for prophylaxis and treatment of haemorrhage or surgical bleeding in patients with VWD when desmopressin is ineffective or contraindicated...Pharmacovigilance data showed low incidence rates for adverse events including FVIII/VWF inhibitors, thromboembolic events, hypersensitivity reactions and transmission of infectious agents. Conclusion This review provides a comprehensive summary of studies that evaluated the use of pdVWF/FVIII in VWD demonstrating the long-term effectiveness and safety of this pdVWF/FVIII across all ages, types of VWD and treatment settings."

Clinical • Review • Cardiovascular • Hemophilia • Immunology • Pediatrics

"You can use iTIVA, using q simulation PK model to stimate residual Heparine and calculate protamine dose" (@deramirezmd)

Immunological risk factors for sepsis-associated delirium and mortality in ICU patients. (PubMed, Front Immunol) - "Nonsurvival critical patients have a distinct immune profile when compared with survival patients. CD14CD16 monocyte prevalence and expression levels of SLC2A1 and STIMATE may be predictors of SAD and 28-day mortality in ICU patients."

Journal • CNS Disorders • Critical care • Immunology • Infectious Disease • Septic Shock • IL6 • SLC2A1 • TNFA

Biochemical and NMR studies reveal specific interaction between STIMATE C-tail and PI(4,5)P or PI(3,4,5)P-containing membrane. (PubMed, Biochem Biophys Res Commun) - "NMR analysis indicates that STIMATE-CT is intrinsically disordered. Furthermore, NMR titration with bicelles and mutation analysis reveal that the regions of VRYR and KKKK in STIMATE-CT are both essential for its membrane binding."

Journal • STIM1

Advances in Intracellular Calcium Signaling Reveal Untapped Targets for Cancer Therapy. (PubMed, Biomedicines) - "We use the PubMed database to highlight current clinical studies that target intracellular Ca signaling. Drug repurposing and improving the delivery of small molecule therapeutics are further discussed as promising strategies for speeding therapeutic development in this area."

Journal • Review • Oncology • CALB1

"In MSD lavoriamo con la speranza di migliorare la sopravvivenza dei pazienti con #tumoredelrene. Nel 2020 sono state circa 13.500 le nuove diagnosi stimate di #carcinomarenale in Italia. #ASCO21" (@msdsalute)

Oncology

"Hi angelo Do you use diaphragm ultrasound to stimate the muscle contraction?" (@pabloeguzman)

Recall on Ferring’s Intranasal Desmopressin Therapies to Affect Availability Into 2021 (Hemophilia News Today) - "A recall on Ferring Pharmaceuticals‘ intranasal desmopressin compounds — used to treat mild to moderate hemophilia A and von Willebrand disease type 1 — is expected to affect the availability of these products until at least mid- to late-2021...Citing higher-than-specified concentrations of desmopressin — a synthetic analogue of vasopressin — in several formulations. The company and other agencies around the world made recall announcements from July 10 to Aug. 5....The recall extends to all lots of DDAVP Nasal Spray 10 mcg/0.1 mL, Desmopressin Acetate Nasal Spray 10 mcg/0.1 mL, and Stimate Nasal Spray 1.5 mg/mL."

Commercial • Hemophilia

Intravenous Paracetamol vs Intranasal Desmopressin for Renal Colic in the Emergency Department: A Randomized Clinical Trial. (PubMed, Pain Med) - "Our study showed that intranasal desmopressin is as effective as intravenous paracetamol for renal colic pain management; however, significant clinical reduction in pain score occurred faster with intranasal desmopressin."

Clinical • Journal • Pain

"DDAVP Nasal Spray 10 mcg/0.1mL, Desmopressin Acetate Nasal Spray 10 mcg/0.1mL, STIMATE Nasal Spray 1.5 mg/mL by Ferring Pharmaceuticals US: Recall - Due to Superpotency https://t.co/ZCAkaBUfTa" (@FDAMedWatch)

Perioperative risk factors of progressive chronic kidney disease following liver transplantation: analyses of a 10-year follow-up single-center cohort. (PubMed, Ann Surg Treat Res) - "Renal function was assessed by the e stimated glomerular filtration rate (eGFR) using the Modification of Diet in Renal Disease formula...More careful renoprotective management is required in elderly LT patients with DM or preexisting proteinuria. Postoperative AKI and vasopressor dose may be potentially modifiable risk factors for progressive CKD."

Clinical • Journal • Acute Kidney Injury • Chronic Kidney Disease • Diabetes • Metabolic Disorders • Nephrology • Renal Disease • Transplantation

[VIRTUAL] Characterization of Type 2B VWD in the US Zimmerman Program (ISTH 2020) - "p.R1341W subjects had a good response to DDAVP/Stimate and no thrombocytopenia... While the majority of type 2B VWD is caused by recurrent SV in the VWF A1 domain, patients with p.R1341W present with a heterogeneous phenotype and mild bleeding symptoms and may be more challenging to diagnose using VWF: RCo/VWF: Ag and multimers alone."

Hematological Disorders • Targeted Protein Degradation • Thrombocytopenia

[VIRTUAL] Moderate Intensity Aerobic Exercise and Intranasal Desmopressin Additively Increase Factor VIII and von Willebrand Factor (VWF) Levels in Adolescent Males with Mild Hemophilia A - Principal Findings from a Randomized Trial (ISTH 2020) - P4 | "While MIA-exercise (compared to desmopressin) was associated with a similar increase in FVIII: C and VWF levels, its impact on hemostasis was brief. Combination of MIA-exercise and desmopressin resulted in an additive increase in multiple hemostatic parameters. These findings have significant implications for persons with MHA."

Clinical • Gene Therapies • Hematological Disorders • Hemophilia • Rare Diseases • F2

Overweight and obesity in Mexican vulnerable population. Results of Ensanut 100k (PubMed, Salud Publica Mex) - "There is a high prevalence of obesity in Mexican population with social limitations and seems to reach the rest of the population quickly but with significant structural disadvantage."

Clinical • Journal • Genetic Disorders • Obesity

[VIRTUAL] Central Diabetes Insipidus in a Patient with Crohn’s Disease (ENDO-I 2020) - "Patient’s hypernatremia and increased urine output started to improve with the switch to IV and was ultimately discharged home with intranasal desmopressin...This case illustrates the importance of considering its presence in the setting of other autoimmune conditions. It also serves to illustrate the complex management and decision making followed in adjusting the desmopressin formulation in the setting of a malabsorptive disease such as Crohn’s."

Clinical • Crohn's disease • Gastroenterology • Immunology • Inflammatory Bowel Disease • Metabolic Disorders • Pain • Rare Diseases • MRI

Self-perceived need for dental treatment and related factors. A cross-sectional population-based study. (PubMed) - Braz Oral Res - "Female adolescents seems to be more sensitive to oral health problems. The results suggest that the DAI score might reflect a self-perceived need for orthodontic treatment and the Angle classification might overestimate the orthodontic treatment need."

Journal • Biosimilar

Proteomic mapping of ER-PM junctions identifies STIMATE as a regulator of Ca(2+) influx. (PubMed) - "Genetic depletion of STIMATE substantially reduces STIM1 puncta formation at ER-PM junctions and suppresses the Ca(2+)-NFAT signalling. Our findings enable further genetic studies to elucidate the function of STIMATE in normal physiology and disease, and set the stage to uncover more uncharted functions of hitherto underexplored ER-PM junctions."

Journal • Biosimilar

Central Diabetes Insipidus in a Patient with Crohn’s Disease (ENDO 2020) - "Patient's hypernatremia and increased urine output started to improve with the switch to IV and was ultimately discharged home with intranasal desmopressin...This case illustrates the importance of considering its presence in the setting of other autoimmune conditions. It also serves to illustrate the complex management and decision making followed in adjusting the desmopressin formulation in the setting of a malabsorptive disease such as Crohn's."

Clinical • MRI