Blueprint Medicines’ Post

The patient voice matters, and Dr. Ashley Herrick, PhD, Executive Director, Oncology Program Strategy, intrinsically understands that. Plus, with more than 13 years of experience in #oncology clinical trial oversight and #drugdevelopment, she knows how much patients, researchers, and sponsors put into these studies – all hoping to change lives for the better. In the upcoming #webinar, “The Voice of the Hematological Malignancy Patient: Are You Listening?”, Dr. Herrick will use her vast experience to share insights about the benefits of patient centricity and how to design patient-centric hematological malignancy trials. Make sure you’ve registered to join this don’t-miss session: https://bit.ly/3SuqxaE #ClinicalTrials #ClinicalResearch #OncologyWebinar #PatientsFirst

🔬 Urgent Need: Rare neuronopathic MPS diseases affect over 1,000 families in the U.S. #RareDisease #NeuronopathicMPS 💡 Biomarker Potential: CSF heparan sulfate offers hope for faster FDA approval of new therapies. #Biomarker #FDAApproval 🤝 Collaborative Drive: Uniting scientific and medical communities to overcome recruitment hurdles and accelerate treatment development. #Collaboration #Research ⏰ Time Critical: With an 8-10 year timeline and half a billion-dollar cost, swift action is vital to save lives. #Urgency #actnow

For those who may have missed it, Dr. David Wirta's article, "The Coming of Age for Dry Eye Disease," provides a comprehensive year-in-review snapshot of the significant progress in Dry Eye Disease (DED) development throughout 2023 and highlights some of Ora's technologies that contributed to this progress.   From insights into FDA-approved therapies to milestone clinical trials, the article emphasizes the role of novel technologies in advancing DED drug development. Whether leveraging remote research solutions, like the Ora EyeCup platform, or decentralized challenge chambers, such as the Ora Controlled Adverse Environment (CAE), 2023 has shown that novel technology and strategies are essential for accessing and enriching global DED patient populations. Explore the full article below to delve into the novel therapies, technologies, and strategies employed in 2023 and beyond! #dryeyedisease #clinicalresearch #ophthalmology

Dr. Alan Tuchman, CEO of Synaptogenix, comments on the safe, significant, and persistent benefits of the 6-month Bryostatin trial for patients with advanced Alzheimer’s Disease. "There is a huge unmet need in the AD population for drugs oriented specifically to advanced and severe patients. Approved drugs do exist for slowing decline in non-demented, Mild Cognitive Impairment (MCI) patients and possible early AD patients, but Bryostatin-1-treated Severe Cohort patients, in contrast, showed no significant cognitive decline across a 10-month span in our study. We believe that Bryostatin-1 for later-stage patients would be complementary to the early-stage AD drugs in providing a full range of treatment options for the more than six million Americans living with Alzheimer's disease." For details, visit: https://prn.to/466Sc5n #ADResearch #Alzheimers

Check out this article on GP-2250 and our #pancreaticcancer clinical trial by Dr. Anup Kasi from University of Kansas Medical Center: https://lnkd.in/etX9hHkp. 

#URGN - UGN102 in Development as the Potential First NonSurgical Therapy for LGIRNMIBC Met Primary Endpoints in Both Phase 3 ATLAS and ENVISION Clinical Trials -- In ATLAS, UGN-102 Demonstrated Superiority to TURBT with a 55% Reduction of Risk for Recurrence, Progression or Death in Patients who Received UGN-102 -- -- ENVISION Showed a Complete Response Rate of 79.2% at 3-Months -- -- Side Effect Profile in ATLAS and ENVISION Con... July 27, 2023 at 09:50AM #StockMarket #MWN

Regarding the number of people on the waiting list for a kidney transplant, approximately 90,000 individuals in the U.S. are on the list, with this number constantly changing due to new patients being added and others receiving transplants or being removed from the list for various reasons. Each year, thousands of patients on the kidney transplant waiting list pass away before receiving a transplant. The exact number can vary year by year, but it's reported that approximately 4,000 to 5,000 individuals may die each year while waiting for a kidney transplant in the U.S. These figures highlight the critical shortage of organ donors relative to the need for transplants. It underscores the importance of organ donation awareness and the impact it can have on saving lives.

In 2008, David Pannick QC,  (now Lord Pannick KC), acting for EISAI in the judicial review of the NICE appraisal of the use of donepezil for the treatment of Alzheimers disease, commented on the lack of stakeholders access to a fully executable* version of the key cost-effectiveness model. He noted that  “the consultee is left making shots in the dark, in circumstances where the light could so easily be switched on.”  Sixteen years later, the highly sophisticated model developed by the PenTAG group as part of NICE’s Renal cell carcinoma Pathways Pilot [ID6186] is publicly available on Github. It is great to see that such open access to these Pathway models is an integral part of the NICE’s Proportionate Approach to technology appraisals initiative https://lnkd.in/dJ4DSRzK. The development of ‘open source’ models in these reviews is a great step forward in transparency  https://lnkd.in/dF5dynwy * It is noted in the judgment that all consultees and commentators were offered a copy of the Model (in ‘read-only’ format).

Carole Ho, Chief Medical Officer & Head of Development at Denali Therapeutics, emphasizes the urgent need for therapies to treat neuronopathic Mucopolysaccharidoses (MPS) and how Denali is focusing its novel technology to on these progressive and debilitating diseases. Read the full piece on BioSpace. ⬇ #RareDiseases #RareDiseaseDay 

🚀 Mesoblast's pioneering MCL technology platform leverages mesenchymal precursor cells to develop tailored therapies for various diseases, promising a paradigm shift in treatment approaches. Mesoblast's allogenic therapy is a strong candidateas treatment for both advanced and end-stage chronic heart failure. Despite challenges, Mesoblast's commitment to innovation extends to inflammatory diseases, exemplified by Ryoncil. Strategic partnerships highlight its relentless pursuit of medical breakthroughs. By modulating the immune system with anti-inflammatory factors, Mesoblast's candidates tackle diseases at their roots, promising transformative outcomes. This approach holds the potential to revolutionize treatment paradigms for conditions like chronic low back pain and beyond. Mesoblast's bold strides toward medical innovation herald a brighter future for patients worldwide. #Mesoblast #MedicalInnovation #newmedicine👩⚕️👨⚕️