Nemours’ Post

Developing drugs for rare diseases poses a range of clinical, regulatory, and commercial challenges. Dive into this curated collection of case studies, webinars, blogs, and whitepapers to gain valuable insights and innovative solutions for developing drugs for rare diseases. http://ow.ly/Zl0i50OvuE9 

Developing drugs for rare diseases poses a range of clinical, regulatory, and commercial challenges. Dive into this curated collection of case studies, webinars, blogs, and whitepapers to gain valuable insights and innovative solutions for developing drugs for rare diseases. http://ow.ly/Zl0i50OvuE9 

Importance:  Endothelin receptor antagonists are first-line therapy for pulmonary arterial hypertension (PAH). The first 2 agents approved in the class, bosentan and ambrisentan, initially carried boxed warnings for hepatotoxicity and required monthly liver function tests (LFTs) as part of a risk evaluation and mitigation strategy (REMS); however, in 2011, as further safety data emerged on ambrisentan, the boxed hepatotoxicity warning and LFT requirements were removed. Conclusions and Relevance:  In this serial cross-sectional study of ambrisentan, labeling changes and removal of the REMS-related LFT requirement were associated with shifts in prescribing and testing behavior for ambrisentan but not bosentan. Further clinician education may be needed to maximize the benefits of REMS programs and labeling warnings designed to ensure the safe administration of high-risk medications.

Putting a child on chronic medication is challenging for parents due to the balance between benefits and potential side-effects, especially given the lack of extensive research on drug safety in children. Many drugs are only approved for adults, leading doctors to prescribe "off-label" based on their judgment, which carries risks. Prescription errors affect about 13% of pediatric patients, occurring more frequently and causing more harm than in adults. Advances in genetic testing, like PharmaGene, offer a way to tailor prescriptions to individual genetic profiles, reducing risks and improving efficacy. Pharmacogenomic testing can identify how a child metabolizes drugs, ensuring appropriate dosing and minimising adverse effects. As this field grows, precision medicine will increasingly benefit pediatric care. Read the full article: https://lnkd.in/dscJ5Njg Find out more about PharmaGene Pharmacogenomics Testing: https://lnkd.in/dn9qEFQH

Stay Informed: Diabetes Mellitus Updates for Primary Care Clinicians Are you a primary care clinician looking to enhance your knowledge on Diabetes Mellitus? Look no further! Explore the latest updates through this comprehensive online CME course. 👩⚕️👨⚕️ Elevate your practice with insights from industry experts and stay ahead in managing Diabetes Mellitus effectively in your primary care setting. This webcast is designed to provide you with the latest research, treatment approaches, and practical tips. 👉Courses included in this package ✅Cardiovascular Disease in Diabetes ✅Focal Segmental Glomerulosclerosis ✅Oral Pharmacology in Type II Diabetes ✅Antidiabetic Pharmacology Part 2 - Injectables ✅Antidiabetic Pharmacology Part 3 Don't miss out on this valuable opportunity! Register now for online CME courses that will take your clinical skills to the next level. Learn more: https://bit.ly/3T3mv9o #DiabetesManagement #PrimaryCare #MedicalEducation #HealthcareUpdates #digitalhealth #Healthcareproviders #CME #CE #eMedEd #MedEd

Our real-world evidence experts have reviewed the latest decision on GAMMAGARD LIQUID. Start reading "FDA Approves GAMMAGARD LIQUID Based on Safety Data From an RWE Study" - https://lnkd.in/et3NiwzY -- Background: The recent approval of GAMMAGARD LIQUID by the FDA for the treatment of Chronic Inflammatory Demyelinating Polyneuropathy (CIDP), is a great example of the power of RWE to inform regulatory decision-making. The approval was based on data from a single-arm Phase III study and a large observational, retrospective comparative safety study assessing the off-label use of GAMMAGARD LIQUID and other intravenous immunoglobulin products for the treatment of patients with CIDP. #RWE #realworldevidence #rwd #realworlddata

𝐂𝐲𝐦𝐚𝐁𝐚𝐲'𝐬 𝐒𝐞𝐥𝐚𝐝𝐞𝐥𝐩𝐚𝐫: 𝐁𝐫𝐞𝐚𝐤𝐭𝐡𝐫𝐨𝐮𝐠𝐡 𝐓𝐫𝐞𝐚𝐭𝐦𝐞𝐧𝐭 𝐟𝐨𝐫 𝐏𝐁𝐂 𝐏𝐮𝐛𝐥𝐢𝐬𝐡𝐞𝐝 𝐢𝐧 𝐍𝐄𝐉𝐌 The New England Journal of Medicine has published detailed results from the RESPONSE Phase 3 trial evaluating seladelpar in adults with primary biliary cholangitis (PBC). The study showed rapid and sustained improvements in reducing cholestasis, liver injury, and pruritus (itching), supporting its potential as a new treatment option. RESPONSE, a double-blind, placebo-controlled trial, demonstrated significant reductions in ALP, ALT, and GGT levels with seladelpar treatment compared to placebo, along with substantial improvements in pruritus scores across multiple scales. Dr. Gideon Hirschfield Gideon Hirschfield, Lily and Terry Horner Chair in Autoimmune Liver Disease Research, Toronto Centre for Liver Disease, emphasized the need for innovative treatments for PBC, noting the substantial benefits observed with seladelpar in the trial. Safety data showed similar profiles between seladelpar and placebo groups, with no significant safety concerns associated with seladelpar treatment. Charles McWherter, Ph.D. Charles McWherter, Ph.D., Chief Scientific Officer and President of Research and Development at CymaBay CymaBay Therapeutics, highlighted the significance of these findings and the potential for seladelpar to transform PBC treatment. With the New Drug Application accepted for priority review by the FDA, and plans for marketing authorization applications in Europe, seladelpar's journey towards regulatory approval is underway. #PBC #NEJM #ClinicalResearch #HealthcareInnovation #LiverDisease #DrugDevelopment #MedicalBreakthroughs

The study investigated the efficacy of sublingual squalene, extracted from pumpkin seed oil, in reducing mortality and re-hospitalization rates among COVID-19 patients. In a randomized clinical trial involving 602 eligible patients, those who received sublingual squalene in addition to standard treatment experienced a significant decrease in mortality compared to those who received standard treatment alone. Additionally, the squalene group had a lower rate of re-hospitalization due to COVID-19. These findings suggest that sublingual squalene in microemulsion form could be an effective drug for improving outcomes in COVID-19 patients. #COVID19Treatment #Squalene #SublingualAdministration #ClinicalTrial #MortalityRate #Rehospitalization #PumpkinSeedOil #MicroemulsionFormulation

The role of plasma angiotensin-converting enzyme and interleukin-6 levels on the prognosis of non-dialysis chronic kidney disease patients. https://ow.ly/sXhf50Qx3tg #Pharmacy #Pharmacology #NaturalProduct #Hypertension

In a randomized clinical trial comparing the efficacy of VV116 versus nirmatrelvir-ritonavir treatment for COVID-19, researchers observed a potential rebound in viral load following treatment. As anti-SARS-CoV-2 drugs become more widely utilized, this study sheds light on the possibility of viral load rebound post-treatment. #COVID19 #ClinicalTrial #ViralLoad 💊🦠🔬 https://lnkd.in/dxTAFrKr