Travere Therapeutics’ Post

Briana Contreras, an editor with Managed Healthcare Executive, spoke with Harsha Rajasimha, MD, founder and executive chairman of Indo US Organization for Rare Diseases (IndoUSrare) in this month's episode of Tuning in to the C-Suite podcast. The conversation was about how the disparity in diversity and ethnicity in rare disease clinical trials in the U.S. has led to gaps in understanding diseases and conditions, jeopardizing universal health and increasing the economic burden of healthcare. #RareDisease #ClinicalTrial https://lnkd.in/gzEqnhjz

Wondering what role DKK3 plays in chronic kidney disease progression? - Then listen to the new ERA podcast with Danilo Fliser, who explains what DKK3 really is and why its measurement helps to better predict the progression of CKD... *Spoiler* (13:51) We can't answer the first question, but we do have an answer for you to the 2nd! Enjoy listening!

Wondering what role DKK3 plays in chronic kidney disease progression? - Then listen to the new ERA podcast with Danilo Fliser, who explains what DKK3 really is and why its measurement helps to better predict the progression of CKD...

#ICYMI: Lindsey Lee Lair, MD, MBA, FAAN, VP of Clinical Development, spoke with Daniel Levine on a Global Genes #RARECast #podcast about #SMA, the progress we’ve seen in treating the condition, and Biohaven’s efforts to develop a therapy to stimulate the growth of muscle mass and strength in people with the disease. Listen to the podcast here: https://brnw.ch/21wGq8V #DaysMatter

In our latest #podcast episode, Panacea Founder and CEO, Dahlia Attia-King, explores the transformative potential of clinical genetic testing, and how Panacea is breaking down barriers and revolutionizing #healthcare accessibility to empower individuals in taking control of their health with disease prevention. Listen in: https://rb.gy/ffwyb0 #financialadvisor #fintech #wealthmanagement #investing

To mark Rare Disease Day 2024, this week we are exploring four critical elements to sustaining rare disease innovation in Europe. Today focuses on requirement #3: Supportive P&R. Whether a medicine exists matters little for patients who cannot receive it because it is not reimbursed. Reimbursement is also key for manufacturers who rely on the sale of medicines to recover their considerable investment in development and to support their future pipeline. Continued innovation and access in rare disease in Europe relies on country-level pricing and reimbursement (P&R) approaches that can recognise and reward promising orphan developments. But this is by no means simple since many extremely novel, transformative treatments fall foul of traditional P&R systems designed for chronic therapies in common conditions. One example is cell and gene therapies. These unique treatments present immense value owing to their one-time, potentially curative profile. They also come with a significant price tag, complex delivery, and an inevitable degree of uncertainty around long-term benefit. In our recent paper for the Alliance for Regenerative Medicines (ARM), Dolon's Managing Director, Adam Hutchings, explores these challenges and their implications for P&R, patient access, and the sustainability of ATMP innovation in Europe. In particular, we focus on innovative contracting as an opportunity to mitigate these challenges as well as recent learnings from manufacturers’ experience in negotiating these. P&R frameworks need to evolve with science if they are to fully reflect the value of emerging therapies to patients and health systems alike. Failure to do so will come at the expense of patients and health systems today, and Europe’s standing as a global centre for innovation in the longer term. Read on to learn more: https://bit.ly/42UC0Uf #RareDiseaseDay2024 #RareDiseaseDay #RDD2024 #LightUpForRare #ShareYourColours #RareDiseases #RareDiseaseAwareness #RareDiseaseInnovation #Dolon

🧬 Curious about the impact of #Genomics on primary care? Tune into our latest episode discussing the high stakes of familial hypercholesterolemia and cystic fibrosis. Learn why knowing your family history is crucial! Hosted by Munir Adam & Claire. #Healthcare #Podcast 🎙️ NHS North Thames Genomic Medicine Service Dominic Studart

NSAIDs work by inhibiting the cyclooxygenase pathway (COX), which is comprised of the COX-1 and COX-2 pathways. The COX-1 pathway is involved in prostaglandin E2–mediated gastric mucosal protection and thromboxane effects on coagulation, while the COX-2 pathway is mainly involved with the modulation of pain and fever without effect on platelet function. While selective COX-2 inhibitors have a decreased side effect profile, all NSAIDS have the potential to cause renal impairment and their use should be limited in patients with underlying renal disease. Learn more high yield-concepts about Anti-inflammatory Medications on our DAILY Orthobullets podcast. Listen wherever you listen to podcasts! https://bit.ly/3L3GQXA

3billion presents 5 research in ASHG'23. Our talk is on how to diagnose rare disease patients of under-represented ethnic groups effectively. 3billion will share the findings and insights on the issue from our experiences of diagnosing ~19,000 ethnically diverse rare patients. Check the schedule below and spare your time! Reviewers' Choice Award-winning research 'Lessons learned from the nationwide 10K rare disease genome project' is another recommendation for you to check out. Talk to our presenters and share insight at ASHG'23

Looking forward to this round table discussions on eliminating Variants of Uncertain Significance (#VUS) and it's impact on #genetic diagnosis and treatments