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Our week starts with a promising piece of news for the #DINcommunity #CIDP #dysimmuneinflammatoryneuropathies #cureDINs #treatDINs #peoplelivingwithDINs #DINpeople #clinicaltrials #medicalresearch

A Phase 3 #clinicaltrial studying the effects of #Ozempic on Type 2 diabetes patients with chronic kidney disease? And, the trial was run during Covid? You have to check this out. https://lnkd.in/gvBmT5wV

Ataxia Telangiectasia (AT) is a rare, progressive disease that typically presents in early childhood and is characterized by neurological and immunological symptoms. Currently, there is no cure or FDA-approved treatment to slow the progression of AT, with the average life expectancy being around 25 years for those diagnosed in childhood. Find out more about ChromaDex's planned development of NRC for the treatment of AT here: https://bit.ly/4cbamFA

Here is a story of a #pharmacist fighting for her kids battling #cysticfibrosis, a disease that impacts the #lungs, and #liver causing #CFLD, cystic fibrosis #liverdisease, #pancreas causing #CFRD, cystic fibrosis-related #diabetes, gut-causing #leakygut, and #dysbiosis. How to diagnose, and treat cystic fibrosis, the #lifeexpectancy in cystic fibrosis, how #pharmacogenomic and #nutrigenomic testing helps and the #functionalmedicine approach to manage #CF. Thank you Julie Wharam, PharmD, CPGx for sharing this story with Healthieyoo! https://lnkd.in/gxyRBpDM

Despite increasing access to new therapies to treat IBD, a paper presented at #ECCO24 tells a story of continued clinical reliance on steroids to control #IBD disease activity. Explore through this explanation of the study presented, the impact increased steroid usage is having on the #IBDCommunity: https://hubs.li/Q02FV0S20 European Federation of Crohn's and Ulcerative Colitis Associations #EFCCA #SteroidToxicity #SteritasGTI

Yet another example of when #realworldevidence #RWE is powerful: when clinical trials are not feasible. We analyzed the effectiveness of the antiviral sotrovimab, when Omicron BA.2 and BA.4/5 variants were most prevalent in 2022, a fast-moving period when setting up a clinical trial was not feasible. We published 2 papers from this work, one on the descriptive characteristics of the patients and another showing our use of inverse probability of treatment weighting to compare outcomes for patients treated with sotrovimab versus untreated patients, in which we show significant reduction of risk of COVID-19 hospitalization for sotrovimab-treated patients aged >65 years and with renal disease. Have a read! 👇 Descriptive data ➡ : https://lnkd.in/dD-Wu9fb Comparative effectiveness ➡ : https://lnkd.in/dWC8CYRf

Despite increasing access to new therapies to treat IBD, a paper presented at #ECCO24 tells a story of continued clinical reliance on steroids to control #IBD disease activity. Explore through this explanation of the study presented, the impact increased steroid usage is having on the #IBDCommunity: https://hubs.li/Q02FTFGW0 European Federation of Crohn's and Ulcerative Colitis Associations #EFCCA #SteroidToxicity #SteritasGTI

As written, the IRA deals a devastating blow to development of treatments for rare diseases. As BIO chair Ted W. Love, MD states in this piece: "Lawmakers can fix this problem right now simply by passing the ORPHAN Cures Act. Introduced by Reps. John Joyce (R-PA) and Wiley Nickel (D-NC) in the House and Sens. John Barrasso (R-WY) and Tom Carper (D-DE) in the Senate, the two-page bill amends the IRA's drug-pricing section by changing the words "only one rare disease" to "one or more rare diseases." With that revision, the legislation vastly increases the odds of treating and curing many of the thousands of rare diseases that currently lack effective medications." This bill has #bipartisan support. Lawmakers MUST do the right thing for patients. We cannot turn our back on patients with rare but devastating diseases. Biotechnology Innovation Organization #patientsfirst #ORPHANCuresACT #rarediseases #orphandrugs #innovation #healthequitynow

#quiztime #rarediseases Rare Disease Quiz: https://lnkd.in/euT74Xc8 I got 22/30. How many did you get? Post in comments. The third panel discussion at the upcoming US Connect, 25–28 February, is 'Are We There Yet? Rare Disease Clinical Trials'. While we may not cover the questions in the quiz, we do intend to address some very interesting ideas: ↳ Global collaboration between researchers, pharmaceuticals and regulatory authorities ↳ Understanding the unique challenges of rare disease clinical trials ↳ Innovations in statistical methodologies that support small population studies Register for the US Connect: https://lnkd.in/ehvjeNNe Full Agenda: https://lnkd.in/eGqjTqYv Panel Discussion on Rare Disease Clinical Trials on 28Feb2024. Meet the Panelists - Sheeba Arnold, Todd Case, Berber Snoeijer, Sophia Zilber 🌺, Michael Litzsinger

Important article in Pharmafile.com by our client Dr Dan Williams at SynaptixBio Ltd. Dan considers the ongoing challenges facing rare disease patients, and discusses developments in the search for therapies. Many thanks to Betsy Goodfellow. The full article is here: https://lnkd.in/e_58twVs.