Taysha Gene Therapies’ Post

Justin Baker, MD, says, “Children should not be viewed as small adults. Like pediatric oncology, the application of palliative medicine in children requires careful consideration and attention to the unique physiologic, psychological, and developmental attributes of each child.” Learn more about a “pallia-paradigm shift” from Dr. Baker, Chief of the Division of Quality of Life and Pediatric Palliative Care and Director of the Quality of Life For All at Stanford Children's Health | Lucile Packard Children's Hospital Stanford: #DaretoBeRare #PatientEmpowerment #HealthcareSolutions #PatientSafety #BeAnythingButOrdinary #LifeWithoutLimits #ChallengeWhatsPossible #PatientEmpowerment #DreamBigWithGusGear #GusGearAcrosstheGlobe [Image Description: Light blue text box against a dark blue background. In white font, “When communicating with and advocating for families, care teams must recognize that young patients are more than their disease.” “Justin Baker, MD, Stanford Medicine Children’s Health.” There is a white Gus Gear logo in a green circle in the upper right corner.] https://bit.ly/3KVQlr2

We are pleased to announce that Alder Hey Children's NHS Foundation Trust and Liverpool Clinical Trials Centre will use the Steritas Pediatric Glucocorticoid Toxicity Index (pGTI) in a clinical trial for Juvenile Idiopathic Arthritis (JIA). The STAR-JIA trial (Steroid TreAtment TRial in JIA) will compare the efficacy, safety and cost-effectiveness of intravenous versus oral glucocorticoids in children with JIA. It will use the Steritas pGTI to systematically assess #SteroidToxicity in children aged 2 to 18. JIA is the most common rheumatic disease in children, yet evidence on optimal dosing and the safety and cost-effectiveness of current first-line treatments remains limited. STAR-JIA trial represents a significant advance in pediatric rheumatology research. Read the full press release here: https://hubs.li/Q02QRlD60 #ClinicalTrials #JIA #JuvenileArthritis #SteritasGTI

🔬 on hepcidin & Fe non-response in pediatric IBD by Dr Bevers 🧒 Non-response to iron therapy in pediatric IBD is high & needs early ID 🔬 Baseline hepcidin better predicts non-response than ferritin or transferrin 📊 New hepcidin cut-offs guide Rx 🔗 https://buff.ly/3ZxMQ2E

We are pleased to announce that Alder Hey Children's NHS Foundation Trust and Liverpool Clinical Trials Centre will use the Steritas Pediatric Glucocorticoid Toxicity Index (pGTI) in a clinical trial for Juvenile Idiopathic Arthritis (JIA). The STAR-JIA trial (Steroid TreAtment TRial in JIA) will compare the efficacy, safety and cost-effectiveness of intravenous versus oral glucocorticoids in children with JIA. It will use the Steritas pGTI to systematically assess #SteroidToxicity in children aged 2 to 18. JIA is the most common rheumatic disease in children, yet evidence on optimal dosing and the safety and cost-effectiveness of current first-line treatments remains limited. STAR-JIA trial represents a significant advance in pediatric rheumatology research. Read the full press release here: https://hubs.li/Q02QR5VH0 #ClinicalTrials #JIA #JuvenileArthritis #SteritasGTI

📣 New paper published: Check out our collaboration outlining the critical need for pediatric screening to detect HoFH in time to begin effective treatment. #KnowHoFH https://lnkd.in/gWZMddNZ

21st- Article Published in Cureus, Indexed in: 1. PubMed 2. PubMed Central 2. WOS Effect of Pediatric Rehabilitation on Children With Viral Encephalitis: A Case Report https://lnkd.in/gMRebWc5

Ophthopedia Update:The incidence of pediatric dacryocystitis among a population-based cohort of infants with congenital nasolacrimal duct obstruction: To report the incidence, clinical characteristics, and outcomes of acute dacryocystitis among a large, population-based cohort of children born with congenital nasolacrimal duct obstruction (CNLDO) over a 10-year period. #Ophthalmology #Eye #Ophthotwitter

📃Scientific paper: Are children with IgA nephropathy different from adult patients? Abstract: Background Previously, several studies have indicated that pediatric IgA nephropathy (IgAN) might be different from adult IgAN, and treatment strategies might be also different between pediatric IgAN and adult IgAN. Methods We analyzed two prospective cohorts established by pediatric and adult nephrologists, respectively. A comprehensive analysis was performed investigating the difference in clinical and pathological characteristics, treatment, and prognosis between children and adults with IgAN. Results A total of 1015 children and 1911 adults with IgAN were eligible for analysis. More frequent gross hematuria (88% vs. 20%, p  < 0.0001) and higher proteinuria (1.8 vs. 1.3 g/d, p  < 0.0001) were seen in children compared to adults. In comparison, the estimated glomerular filtration rate (eGFR) was lower in adults (80.4 vs. 163 ml/min/1.73 m^2, p  < 0.0001). Hypertension was more prevalent in adult patients. Pathologically, a higher proportion of M1 was revealed (62% vs. 39%, p  < 0.0001) in children than in adults. S1 (62% vs. 28%, p  < 0.0001) and T1–2 (34% vs. 8%, p  < 0.0001) were more frequent in adults. Adjusted by proteinuria, eGFR, and hypertension, children were more likely to be treated with glucocorticoids than adults (87% vs. 45%, p  < 0.0001). After propensity score matching, in IgAN with proteinuria > 1 g/d, children treated with steroids were 1.87 (95% CI 1.16–3.02, p  = 0.01) times more likely to reach complete remission of proteinuria compared with... Continued on ES/IODE ➡️ https://etcse.fr/i4AC ------- If you find this interesting, feel free to follow, comment and share. We need your help to enhance our visibility, so that our platform continues to serve you.

See my latest publication in Pediatric Reports (IF= 1.4)

Do you have or know a child aged 6-17 years old with Cushing's disease? UCSF is conducting a trial looking at osilodrostat (Isturisa) in children and adolescent patients with Cushing’s disease. Isturisa is currently FDA-approved for adults, and works by blocking an enzyme that helps make cortisol leading to less cortisol in the body.  Families who participate in clinical trials and other research studies contribute to science and learn more about their child's condition under top doctors who run those trials, and findings from the research help future patients as well. Most trials for medications, including this one, also offer extension periods after the initial trial if the patient does well on the medication being studied and wishes to stay on it. Trial sponsors do everything they can to lessen the burden of participation including covering expenses like travel, meals, and lodging, and they will sometimes offer financial compensation for participation. This trial offers all of the above!   To learn more about this study being conducted at the University of California - San Francisco (UCSF), please contact Luis Gay, MPH, UCSF Pediatric Endocrinology, at 415-990-7296 or luis.gay@ucsf.edu https://lnkd.in/g2UN_EVS #cushings