X4 Pharmaceuticals’ Post

😊 Great day at The Pain Research and Therapeutics Conference this week, hosted by OBN at Reading University. Fascinating presentations by Rich Harrison, Weibke Gahndi, Amelia Hollywood and Dr Deepak Ravindran. Love the holistic approach. Pain meds are not the whole answer! #holisticpainrelief #HolisticPainManagement #myofascialrelease #HolisticHealth #myofascialtherapy #myofascialreleasetherapy #HopeAndHealing #chronicfatigue #PainManagement

This May is Friedreich Ataxia Awareness Month. Redenlab is a global leader in speech and swallowing clinical endpoints for Friedreich Ataxia, having supported more than two dozen clinical trials and natural history studies in the condition, including UNIFAI, the global study of FA. Most people with Friedreich ataxia are deeply concerned about communication, rating speech as a top priority for treatment (FDA Externally-led Patient-Focused Drug Development Meeting, 2017). Dysarthria and dysphonia are common in hereditary ataxia and worsen over time, affecting all speech subsystems and making speech slow, unclear, and hard to understand. Redenlab's speech tests utilise objective speech markers to track changes in disease and communication, providing valuable data for evaluating therapy efficacy and enhancing clinical trial endpoints. For more information, please visit https://lnkd.in/gEiXEVeR. #Research #FreidriechAtaxia #Ataxia #Study #Speech

Last Chance! This webinar intends to improve knowledge of the role of bronchoscopic ablative treatments & their benefits/limitations for lung malignancies & gain insight into future developments in this rapidly growing therapeutic approach. Register: https://bit.ly/BAT4LM #LCSM

#HappeningNow: In partnership with Incyte, we’re presenting the full results from our AGAVE-201 pivotal trial of axatilimab in patients with chronic GVHD during American Society of Hematology’s #ASH23 Plenary Scientific Session.   We believe that the additional positive data from AGAVE-201 further strengthen axatilimab’s strong safety and efficacy profile as a well-differentiated treatment option for patients with refractory chronic GVHD.   Read the full press release and view the additional data here:  https://bit.ly/3RBczCQ

In this phase I trial, 114 subjects were randomized, 55 (mean age 29 years, 64% female) assigned to the single ascending dose period and 59 (mean age 32 years, 58% female) assigned to the multiple-ascending dose period, of whom 105 completed the trial. The results showed that muvalaplin reduced lipoprotein(a) [Lp(a)] levels by up to 65% without any safety concerns, providing a basis for further clinical development. Learn more about the results of this study on our website. ➡️ https://lnkd.in/eqtcRUfP #R3i #Lipoprotein #Cardiovascular #CardiovascularHealth #CVD #CardiovascularDisease #ResidualCardiovascularRisk #Muvalaplin

The efficacy and safety of a fixed-dose combination of apocynin and paeonol, APPA, in symptomatic knee OA: A double-blind, randomized, placebo-controlled, clinical trial https://bit.ly/3xbI6nN #Osteoarthritis #OA #KneeOsteoarthritis

Delve into the intricacies of Paroxysmal Nocturnal Hemoglobinuria (PNH) through our latest published article. We unravel a rare complication, Popliteal Artery Thrombosis, shedding light on its clinical manifestations, diagnostic challenges, and therapeutic interventions. This insightful case report offers valuable insights for clinicians and researchers alike, aiming to enhance our understanding and management of PNH-related complications. #PNH #Thrombosis #MedicalResearch

Asking the right questions during primary research is critical to understanding future treatment algorithms and competitive landscapes in any disease area. Earlier this week, I read several articles on the positive news for Eli Lilly and Company's donanemab from the FDA advisory panel. Still, these were the questions I was left with: 1. If amyloid plaque is cleared below the threshold and treatment stopped, will the slowing of disease progression continue at the same rate in the long term? 2. Will amyloid begin to reaccumulate longer term (post 12 months)? Will it be faster or slower than the first time? 3. How will you monitor amyloid longer-term post-treatment in the real world? 4. Is there the possibility that a second treatment regimen will be needed if amyloid reaccumulates? When would you start this? Realistically, many of these answers will only come from the collection of longer-term real-world data, which, hopefully, the FDA will advise Lilly to conduct as part of any approval. However, most KOLs or those working in the space could posit answers that would help anyone working in this space understand how changes may happen in the future. Regardless, it is exciting that we may have a second disease-modifying therapy for Alzheimer’s after many years, which is nothing but symptomatic treatment. #alzheimer #lifescience #primaryresearch

Mom didn't know the drug wouldn't work for her. The doctor just prescribed "the protocol". One size does NOT fit all. After Mom's cancer was removed, she was prescribed tamoxifen per the 5-year protocol. Back in 2007, no genetic testing was done to determine drug compatibility. When I ran a Pharmacogenomic (PGx) test using her DNA in 2022, we learned that her body did not break down tamoxifen efficiently and she received NO benefit from the drug. I would definitely recommend a test for anyone who has not already been tested. It could be difference between life and death. And if you have been tested, ask for the results and share them with ALL your medical providers. Keep a copy of the results on your phone and/or in your medical notebook. If you don't understand your results, I can help with that! www.qrxconsults.com

New data to help in treatment decisions for Crohn's disease patients! A study recently published in the New England Journal of Medicine shows that risankizumab, brand name SKYRIZI® is more effective than ustekinumab, brand name STELARA® in achieving clinical and endoscopic remission in patients with moderate-to-severe Crohn’s disease who had unacceptable side effects with anti-TNF therapy or an inadequate response to such therapy. Significant strides in treatment options! Find the clinical evidence for both #SKYRIZI & #STELARA on DRUGDOCS® Read the paper here: https://lnkd.in/ex5znVzp #CrohnsDisease #MedicalResearch #IBD