QurAlis的动态

This week we advanced QRL-201, our ASO candidate targeted at restoring STMN2 expression in ALS, to the dose range finding stage of our ANQUR study. This will include additional biomarkers of disease progression. #STMN2 #ALS #precisionmedicine

? Exciting progress from QurAlis! ?? Thrilled to see the ANQUR clinical trial for QRL-201 advancing to the dose range-finding phase. ?? With its potential to restore STMN2 expression and target disease-modifying outcomes in ALS, including C9orf72-related ALS, this work represents a significant step forward in precision medicine for neurodegenerative diseases. ?? Kudos to the team for their dedication to bringing innovative therapies closer to patients worldwide! ??

Insilico Medicine has announced the completion of enrollment for its Phase IIa clinical trial (NCT05938920) of the novel small molecule inhibitor INS018_055 in China. This study aims to evaluate the safety, tolerability, and preliminary efficacy of INS018_055 in treating patients with idiopathic pulmonary fibrosis (IPF). The randomized, double-blind, placebo-controlled study is underway across 29 research centers in China, with 71 patients enrolled as planned. In addition, Insilico Medicine is preparing for a Phase IIb proof-of-concept study scheduled to commence in 2025 to further explore the efficacy and safety profile of INS018_055. IPF is a chronic, progressive lung disease affecting approximately 5 million people globally. Diagnosis often occurs in advanced stages, with a median survival of only 3 years post-diagnosis. Current anti-fibrotic medications may slow disease progression but often fail to halt or reverse its course, and they are associated with significant side effects impacting patient quality of life. #MedicalInnovation #IPF #ClinicalTrial #HealthcareResearch #QimingPortfolio #QimingHealthcare

The ASC4FIRST trial was presented at ASCO, comparing asciminib to first and second generation TKIs in newly diagnosed CML.? Asciminib demonstrated superior MMR rates and side effect profiles compared to first and second generation TKIs.? In a subgroup analysis, this difference was statistically significant when compared to imatinib, and was not statistically significant when compared to the second generation TKIs, although asciminib was still numerically better.? What's more important to note, however, is that other outcomes like progression-free survival and overall survival have not read out yet.? Examining the side effect data, we can probably say that asciminib is better tolerated than the BCR-ABL TKIs.? Every TKI has its "signature" side effect, like edema or arterial occlusive events, which clinicians have to learn to manage.? In this trial, asciminib had 38% grade 3 or higher adverse events, compared to 44% for imatinib and 55% for the other TKIs.? From a clinical outcome perspective, it's not time to move to asciminib, but it may be a reasonable first line option for certain patients from a side effect standpoint. #scemblix #cml https://lnkd.in/eq3QNtRc https://lnkd.in/ekqxfjGM

I am a little late to this because this good news happened to coincide with the birth of my daughter and so it understandably was a bit overshadowed at the time. But now that I’ve had a little time to get my dad legs under me I am tremendously proud to share that the results of a multi-year effort to study the real-world effectiveness of Vertex’s Trikafta drug were recently published in Heliyon, a Cell Press publication. This was a study in which my team worked with 15 hospitals to extract and normalize data for a population of 206 cystic fibrosis patients.? The study measured a variety of outcomes including lung function, pulmonary exacerbations, BMI, and blood glucose levels. The objective of the study was to measure the effectiveness of Trikafta in improving these outcomes (results that were demonstrated in Vertex’s clinical trials of the drug) and the study supported clinical trial results and demonstrated the effectiveness of Trikafta in a real-world setting. Reinforcing clinical trial results through the use of electronic medical records recorded during the routine course of patient care (i.e. with no additional manual data entry into an EDC through eCRFs) is no simple feat, and something that I feel personally thrilled to have accomplished. Hopefully this paper can provide supporting evidence when making market access decisions for this beneficial drug. Many people were involved in the publication of this paper but I want to give a special shout out to the folks on my team - Brittany McEvoy and Anna Cole Crosbie (former OMie) - for all of their hard work on the data sourcing, quality control, and standardization aspects of this project. You can read the full article at the link below. https://lnkd.in/eBaCGfxA

The impact of institutional measures on optimal use of intravenous immunoglobulin Jean-Nicolas Champagne1 | Antoine Desilets 2 | Guillaume Roy1 | Océane Landon-Cardinal 3 | Hugo Chapdelaine4 | Geneviève Matte5 | Claudia Bouchard6 | Benjamin Rioux-Massé2,7 | Anne-Sophie Lemay2,7 Transfusion. 2024;1–10. https://lnkd.in/gPzdDwVT? Intravenous immunoglobulin (IVIG) shortage represents an emerging issue in transfusion medicine. Limited data are available to determine effective strategies for optimal use. The objective of this retrospective observational study was to determine the impact of institutional measures on IVIG use at a large academic center. In conclusion, a global reduction in IVIG use and a preferential decrease in the unrecommended orders of IVIG were observed in this retrospective study. While nonrestrictive measures initially led to a modest reduction, the implementation of restrictive measures resulted in a more substantial and sustained decrease in IVIG use. These changes were likely due to the bundle of strategies employed, including mandatory use of IVIG order forms, blood bank gatekeeping, and the IVIG stewardship subcommittee. The study confirms the effectiveness of these methods, though the magnitude of the impact depends on the initial level of ordering appropriateness.

Excited to share my recently published research! Imagine living every day knowing your red blood cells could be destroyed at any moment. This is the reality for patients with Paroxysmal Nocturnal Hemoglobinuria (PNH), a rare and life-threatening blood disorder caused by uncontrolled hemolysis. My research, titled "Iptacopan Efficacy and Safety to Treat Paroxysmal Nocturnal Hemoglobinuria (PNH): A Systematic Review and Meta-Analysis" , explores the potential of Iptacopan as a groundbreaking treatment. By targeting factor B and disrupting the complement pathway earlier, Iptacopan offers hope for better outcomes with the convenience of oral administration—unlike traditional intravenous therapies. Special thanks to Dr. Ibrahim M. Dighriri and my colleagues : ? Reham M. Al-Qahtani ? Amal O. Almutairi ? Rahaf N. Alhashbari ? Hanin A. Kanbeja ? Salman A. AlOjaimi ? Mona A. Aljuaid ? Abdullah A. Albaradi ? Sahar T. Almanjumi ? Samah A. Alqurashi ? Maryam S. Majrashi ? Hatoon M. Alansari ?Abdulaziz S. Alharbi ? Amnah A. Alnami If you're interested in learning more, you can read the full study here: https://lnkd.in/e2bhwEsZ #PNH #Iptacopan #Research #Healthcare #Pharmacy #BloodDisorders #ClinicalPharmacy #MedicalResearch #PatientCare

Substantial clinical evidence links CSF heparan sulfate to clinical outcomes in?neuronopathic?#MPS patients and the FDA recently accepted the biomarker to support #AcceleratedApproval. A workshop hosted by the Reagan-Udall Foundation for the FDA earlier this year brought together industry, researchers, and advocates to discuss this evidence - and the speakers have now published a summary of their perspective on the path forward for neuronopathic MPS disorders. Learn more: https://lnkd.in/dNA49ThV #RareDisease

PREMIER trial @ESC Congress 2024 PIONEER-HF and PARAGLIDE-HF demonstrated that early initiation of sacubitril/valsartan (Sac/Val) led to a greater reduction in N-terminal pro-B-type natriuretic peptide (NT-proBNP) levels in patients stabilized after AHF, especially in those with a left ventricular ejection fraction (LVEF) below normal. These studies were conducted in North America and included few Asian patients.?Generally, there are substantial differences between the USA and Japan in terms of medical systems, clinical practice, and outcomes of patients with AHF; hence, it is clinically relevant to examine whether the current evidence on Sac/Val therapy in patients with AHF can expand outside North America. Therefore, the Program Angiotensin-Neprilysin Inhibition in Admitted Patients with Worsening Heart Failure (PREMIER) study was conducted to assess the efficacy and safety of in-hospital Sac/Val therapy initiation after an AHF event in the Japanese clinical setting. In-hospital Sac/Val therapy initiation in addition to contemporary recommended therapy triggered a greater NT-proBNP level reduction in Japanese patients hospitalized for AHF. These findings may expand the evidence on Sac/Val therapy in this clinical situation outside North America. https://lnkd.in/gSttPjc8 #agposts #conferenceupdates #clinicaltrial #clinicalstudy #cardioupdate #cardiology #heartfailure #Cardiovascular ?

Learn how PRO measures can support the study and development of amyloidosis treatments through clinical trials, observational studies and other applications:https://lnkd.in/gQWkyk3Y #COAs #clinicaltrials #PROMs