News and Trends 14 Nov 2022 A new treatment for tackling epidermolysis bullosa By Jordi Casals, president, EMEA, Amryt Pharma Epidermolysis bullosa (EB), sometimes described as “the worst disease you’ve never heard of,” is a group of rare and distressing inherited skin disorders primarily diagnosed in young children. The genetic mutations that cause EB can result in the skin being extremely fragile. Any physical contact with the skin […] November 14, 2022 - 3 minutesmins - By External Contributor Share WhatsApp Twitter Linkedin Email
News and Trends 14 Nov 2022 Altered cell behavior reason for neuroblastoma resistance Researchers at Lund University in Sweden have identified one of the reasons why the childhood cancer neuroblastoma becomes resistant to chemotherapy. The scientists hope the findings are significant for how future treatments should be designed. The results have been published in the journal Science Advances. Neuroblastoma is an aggressive cancer of the sympathetic nervous system, […] November 14, 2022 - 2 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 14 Nov 2022 New therapy transitions toxic immune cells from harmful to healing to fight effects of hyperinflammation in children When we think about acute kidney injury (AKI) and multi-organ failure, we don’t often think about children. But approximately 4,000 pediatric patients in the U.S. each year have AKI requiring continual kidney replacement therapy (CKRT). These patient profiles are associated with long hospital stays, high morbidity and mortality (50%), and children who survive an AKI […] November 14, 2022 - 5 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 14 Nov 2022 InfanDx tackles early detection of hypoxic ischemic encephalopathy InfanDx AG, a privately held diagnostics company focusing on the development and commercialization of novel diagnostic solutions for newborns, has completed the validation of metabolic biomarker panels for the early detection of hypoxic ischemic encephalopathy (HIE) in newborns. The validated biomarkers of such panels will be the target of the company’s HypoxE blood test for […] November 14, 2022 - 8 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 14 Nov 2022 RSV cases are rising fast in children. Early detection can help. By Shawn Gaskell, MilliporeSigma RSV-related hospitalizations for 2022 have already surged to unusually high numbers, surpassing cumulative cases typically reported in late December each year, and we’re not even one month into the season. While no age group is immune to RSV— respiratory syncytial virus— and the trouble it can cause, young children are being […] November 14, 2022 - 5 minutesmins - By External Contributor Share WhatsApp Twitter Linkedin Email
Interview 14 Nov 2022 In depth interview: AstraZeneca/Sanofi RSV drug Beyfortus AstraZeneca and Sanofi’s Beyfortus (nirsevimab) has been approved in the European Union (EU) for the prevention of respiratory syncytial virus (RSV) lower respiratory tract disease in newborns and infants during their first RSV season. Beyfortus is the first and only single-dose RSV passive immunization for the broad infant population, including those born healthy, at term […] November 14, 2022 - 9 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 14 Nov 2022 Early diagnosis tool for childhood kidney disease Early diagnosis of chronic kidney disease (CKD) is key to managing progression of the disease. A new technique analyzing urine extracellular vesicles (uEVs) — cell-derived nanoscale spherical structures involved in multiple biological functions — in urine samples, identifies changes in the kidneys earlier than conventional methods and can also predict renal function decline. A team […] November 14, 2022 - 4 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 14 Nov 2022 A biotech boom manifesting real results in rare pediatric diseases By Bob Coughlin, managing director, life sciences, JLL For the last two years, the pandemic has resulted in the explosive growth in life sciences. The world watched in awe and anticipation as biotech successfully raced to develop vaccines, therapeutics and diagnostics for Covid-19. It has been a good moment for an industry that deserves such […] November 14, 2022 - 5 minutesmins - By External Contributor Share WhatsApp Twitter Linkedin Email
News and Trends 14 Nov 2022 Researchers discover protein facilitates relapse of pediatric brain tumors The malignant brain tumor type medulloblastoma can become resistant to therapy, which can cause relapse. Researchers at Uppsala University in Sweden have discovered a protein that makes tumor cells insensitive to radiation treatment. The scientists hope the results could lead to better treatments for children with the highest risk to develop relapses. Avoiding treatment Medulloblastoma […] November 14, 2022 - 3 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 11 Nov 2022 Prurigo nodularis treatment recommended for EU approval The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion, recommending the approval of Dupixent (dupilumab) in the European Union (EU) to treat adults with moderate-to-severe prurigo nodularis who are candidates for systemic therapy. Dupilumab is being jointly developed by Sanofi and Regeneron. The European Commission is expected […] November 11, 2022 - 3 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 11 Nov 2022 PACT Pharma reports data from CRISPR cancer study PACT Pharma, Inc. has reported data from the first clinical study using CRISPR gene editing technology to substitute a gene in patients’ immune cells to treat cancer. Results from the study, which was conducted with collaborators at nine academic centers using PACT Pharma’s platforms, demonstrated early proof-of-concept that a patient’s immune system can be reprogrammed […] November 11, 2022 - 5 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 11 Nov 2022 Program management support: accelerating growth for early stage biotechs More academic groups, new start-ups, biotechs and small pharma companies are turning to external consultants for professional program management support and guidance to help them progress their ideas into medicines for patients. Ian Hollingsworth, associate director of product development at Boyds, a global drug development consultancy, explains the common reasons why academic spinouts and small […] November 11, 2022 - 6 minutesmins - By External Contributor Share WhatsApp Twitter Linkedin Email