Mironid® Limited’s Post

Recent Research Advances of Fabry Disease - Pathogenesis   Dear readers, We would like to share a nice Review with you "Neuropathy and pain in Fabry disease " https://lnkd.in/gZKxKunJ (click to download the PDF for free) from Dr. Nurcan Üçeyler, which we hope it will be helpful and useful to your research. Our journal welcomes high-quality articles, we sincerely invite you to contribute an article related to rare disease or orphan drugs to our journal. To fully support our authors to publishing high quality articles in our journal, the invited articles will undergo standard peer review process and enjoy fast publishing service.   About RDODJ: https://lnkd.in/g6_xPjbs   Rare Disease and Orphan Drugs Journal always welcome your submissions. #rare disease #orphan drugs #Fabry disease

The first application of an off-the-shelf product has been reported to be effective in a few patients.In efforts to treat refractory autoimmune diseases, patient T cells have been engineered to target CD-19. Now TyU19 is a genetically engineered, healthy donor-derived CD19-targeting CAR-T product TyU19 caused B cell depletion in three patients with refractory autoimmune diseases TyU19 alleviated severe skeletal muscle damage in a patient with refractory IMNM TyU19 reversed extensive fibrotic damage to critical organs in two patients with dcSSc https://lnkd.in/gGuse_ve

Did you know that there are over 6000 unique rare diseases, with almost 72% of them being genetic and almost 70% exclusively affecting children? Shockingly, for a subset of these rare diseases, it is estimated that between 263 to 446 million people worldwide have one of them - that's about 1 in 17 people! Exciting news from Stoke Therapeutics as they recently released Phase I/II data showing that in combination with anti-seizure medicines, nine patients in the study had a 74% reduction in seizures at six months after the last dose. Read more about it here: https://lnkd.in/em8UX6Wz While it is still early days, the company is planning a registration trial. Stay tuned for more updates on how Stoke Therapeutics is making strides in the treatment of rare diseases. Interesting links: Clinical Trials website detailing the clinical trial: https://lnkd.in/eGCixS4R Rare Disease Emerging as a Global Public Health Priority https://lnkd.in/eH-xCBza). Estimating cumulative point prevalence of rare diseases: analysis of the Orphanet database https://lnkd.in/eQ6vkA6B #GeneTherapy #DravetSyndrome #FDA #Biotech #RareDisease

🎗️ Today, April 24th, we join in observing National Cystic Fibrosis Day, a day that reminds us of the daily challenges faced by patients and their families. This chronic disease not only requires considerable dedication in terms of treatment but also carries a significant emotional burden, increasing anxiety and depression rates among these patients. At BDR-Blackhills Diagnostic Resources, we are committed to innovation and supporting the medical community and patients dealing with this condition. That’s why today we want to highlight our Genvinset® deltaF508 diagnostic kit, an essential tool for the efficient detection of the deltaF508 mutation in the CFTR gene, the most common in Cystic Fibrosis cases. With the advent of innovative treatments and new CFTR modulators, and additional challenges like the recent COVID-19 pandemic, the ability to adapt and overcome new challenges is more crucial than ever. Our Genvinset® deltaF508 kit is a step forward in this adaptation, offering quick and reliable results that aid in critical clinical decision-making. For more information about our kit and how it can facilitate the diagnosis of Cystic Fibrosis, visit our website. #CysticFibrosis #Health #Diagnostics #HealthInnovation 💙

Recent Research Advances of Fabry Disease - Pathogenesis   💜 Dear readers, We would like to share a nice Review with you "Investigating Fabry disease - some lessons learned" https://lnkd.in/gq7Jg4nv (click to download the PDF for free) from Dr. Raphael Schiffmann, which we hope it will be helpful and useful to your research. 🤝 Our journal welcomes high-quality articles, we sincerely invite you to contribute an article related to rare disease or orphan drugs to our journal. To fully support our authors to publishing high quality articles in our journal, the invited articles will undergo standard peer review process and enjoy fast publishing service.   About RDODJ: https://lnkd.in/g6_xPjbs   Rare Disease and Orphan Drugs Journal always welcome your submissions. #Fabry #Fabry disease #Raphael Schiffmann #rare disease #Therapy

Among patients with polyarticular-course juvenile idiopathic arthritis who started receiving a second biologic, there is no difference in achievement of inactive disease or minimal disease activity at six months between receiving a second second tumor necrosis factor inhibitor (TNFi) versus a non-TNFi biologic. The most common biological treatment scheme in these patients is to use #etanercept as the first biological (84%), and after discontinuation of receiving a first TNFi to switch to a second TNFi (85%), usually #adalimumab, rather than change to a different class of biologic. 🔗https://lnkd.in/dVGcN8FF

Daniele Noviello presents this study focusing on the impact of different biological therapies on a subset of Th17 cells, known as pathogenic Th17 (pTh17) cells, in patients with Crohn’s disease. The research demonstrates that Risankizumab, unlike Ustekinumab or Vedolizumab, effectively downregulates IL-23-induced mucosal pTh17 cells in these patients. #Crohns #ECCO24 #Risankizumab

🌟 New paper alert 🌟 Exploring the role of eosinophils in IBD! 🔬 #IBD #Eosinophils Read more: https://lnkd.in/eS3gvh3Y

Daniele Noviello presents this study focusing on the impact of different biological therapies on a subset of Th17 cells, known as pathogenic Th17 (pTh17) cells, in patients with Crohn’s disease. The research demonstrates that Risankizumab, unlike Ustekinumab or Vedolizumab, effectively downregulates IL-23-induced mucosal pTh17 cells in these patients. #Crohns #ECCO24 #Risankizumab

Daniele Noviello presents this study focusing on the impact of different biological therapies on a subset of Th17 cells, known as pathogenic Th17 (pTh17) cells, in patients with Crohn’s disease. The research demonstrates that Risankizumab, unlike Ustekinumab or Vedolizumab, effectively downregulates IL-23-induced mucosal pTh17 cells in these patients. #Crohns #ECCO24 #Risankizumab