Harrington Discovery Institute at University Hospitals ’s Post

A novel and disruptive approach to conducting clinical research in patients with rare diseases. This is intriguing! The Scleroderma Research Foundation's CONQUEST trial is an innovative and potentially disruptive research platform that provides multiple developers with the ability to enroll and execute a single Phase 2b study. This approach could substantially decrease the time and cost of executing studies initial PoC compared to traditional approaches while accelerating the development of much-needed products for rare and debilitating diseases...

The saying goes, "If you're awarded one NIH grant, your organization and it's corresponding program is really on to something... when you're awarded your second NIH grant for the same program, you could change the world!" 🌎 Ok, I just made up that "saying", but it is true 😁 Congratulations to Urban A. Kiernan, Ph.D. and iMetabolic Biopharma Corporation for this incredible achievement that very few biotech/TechBio's ever realize. This Phase 1 grant (1R43HL174270) is funded by the National Heart, Lung, and Blood Institute (NHLBI) for the further development of iMBP's innovative drug program in the treatment of atherosclerotic cardiovascular disease (ASCVD), the underlying pathogenic driver leading to heart attack and stroke. In the United States, ASCVD afflicts about 26 million people, and results in 2 million hospitalizations and 400,000 deaths every year (PMID: 35430874). iMBP is creating programs in parallel via it's proprietary iPlatform™ that could mean drastic advancement in neurodegenerative diseases like Alzheimer's disease as well. Please follow iMetabolic Biopharma Corporation and Dr. Kiernan's LinkedIn pages for the latest in these innovations. Check out the full Yahoo! Finance press release below... 📸 Credit: Maricris Lapaix Hyland (in my iMBP rain jacket on a stormy day in Japan July, 2024) https://lnkd.in/g6jTi56s

Why was #dexamethasone life-saving for severe #COVID? Single-cell omics meets precision medicine in this Cell paper from Anna Aschenbrenner and colleagues to inform us why.

The renewal of the Rare Pediatric Disease Priority Review Voucher (PPRV) must be a top priority for Congress. MassBio signed the Council of State Bioscience Associations (CSBA) letter calling for its reauthorization earlier this month. Thank you, Leslie J Williams, for bringing these issues to the forefront with this excellent op-ed in the BBJ about how PPRV helps address the economics of rare-disease research. Leslie writes, "If the PPRV program isn't renewed, it could threaten progress not just for the biotech industry, but for the more than one million American children suffering from rare diseases. For these kids and their families, the PPRV program isn't just abstract policy — it's a beacon of hope for a healthier future." Just a few weeks ago I was with Leslie at HC Bioscience, Inc. with an incredible 10-year-old from Texas with Duchenne Muscular Dystrophy. He was scientist for a day thanks to Make-A-Wish. They even had business cards made up for him, and I have one on my desk as a daily reminder. Congress must do no harm when it comes to patients, and renewing PPRV is essential. https://lnkd.in/eNGEu8kk #massbiomember #PPRV #raredisease #patientdriven

👏 Special Issue “Advances in Neurofibromatosis - The Future is Bright” from Rare Disease and Orphan Drugs Journal. Prof. Allan E. Rubenstein and Dr. Gurcharanjeet Kaur invite you to contribute to us! 💬 This Special Issue will help to understand the landscape, current advances, and remaining challenges for the development of Neurofibromatosis. We invite manuscripts that cover a wide range of relevant topics. More details about the Special Issue, please click https://lnkd.in/g3kvRQfU 💡 Here is the submission information for authors to refer to For Author Instructions, please refer to https://lnkd.in/gfpYEwyx For Manuscript Templates, please refer to https://lnkd.in/ggymCDPP For Online Submission, please login at https://lnkd.in/gqUwPJu5 If you have any questions, please contact in-house Editor Stella of RDOD Journal Office, assistant_editor@rdodjournal.com. Welcome contributions from experts in this field! #Neurofibromatosis #rare disease #orphan drug

Single-cell analysis decodes the mechanisms of dexamethasone in #COVID-19

What great news to see. Very excited to see how this partnership will allow the NHS laboratory team to continue their translational research activity and how this will impact the delivery of highly specialised diagnostics to mitochondrial disease patients.

Make an impact in the search for a cure to scleroderma. 🔬 Help advance scleroderma research by participating in research studies. Your participation helps researchers better understand the unique ways this disease affects people and brings us closer to a world without scleroderma. Here’s how you can get involved: 🔹 Join the CONQUER Registry: The CONQUER Registry is a first-of-its-kind nationwide patient registry and biosample repository aimed at improving care and developing more effective, personalized therapies for SSc patients. 🔹 Consider enrolling in CONQUEST: The CONQUEST study is a scleroderma clinical trial for people with systemic sclerosis associated interstitial lung disease (SSc-ILD). Participants help progress research to potentially uncover new SSc-ILD treatment options. 🔹 Explore clinical trials: Clinical trials are research studies in which people volunteer to help find answers to specific health questions. Joining a clinical trial is a meaningful way to get involved with research and help find a cure. Learn more about how you can participate in innovative research that aims to improve therapies and ultimately, find a cure: https://bit.ly/3WtHOCd

SYMPOSIUM ABOUT FOP Are you working with the very rare bone condition Fibrodysplasia Ossificans Progressiva (FOP) as a professionals or patient organization? Check out this symposium organized by the Rare Bone Disease Alliance in the USA. Please share this information with people you think it could be relevant for. #rareboneconditions #FOP

📃Scientific paper: HSV-1 cellular model reveals links between aggresome formation and early step of Alzheimer's disease Abstract: International audience; Many studies highlight the potential link between the chronic degenerative Alzheimer's disease and the infection by the herpes simplex virus type-1 (HSV-1). However, the molecular mechanisms making possible this HSV-1-dependent process remain to be understood. Using neuronal cells expressing the wild type form of amyloid precursor protein (APP) infected by HSV-1, we characterized a representative cellular model of the early stage of the sporadic form of the disease and unraveled a molecular mechanism sustaining this HSV-1- Alzheimer's disease interplay. Here, we show that HSV-1 induces caspase-dependent production of the 42 amino-acid long amyloid peptide (A beta 42) oligomers followed by their accumulation in neuronal cells. A beta 42 oligomers and activated caspase 3 (casp3A) concentrate into intracytoplasmic structures observed in Alzheimer's disease neuronal cells called aggresomes. This casp3A accumulation in aggresomes during HSV-1 infection limits the execution of apoptosis until its term, similarly to an abortosis-like event occurring in Alzheimer's disease neuronal cells patients. Indeed, this particular HSV-1 driven cellular context, representative of early stages of the disease, sustains a failed apoptosis mechanism that could explain the chronic amplification of A beta 42 production characteristic of Alzheimer's disease patients. Finally, we show that combination of flurbiprofen, a non-steroidal anti-inflammatory drug... Continued on ES/IODE ➡️ https://etcse.fr/16n ------- If you find this interesting, feel free to follow, comment and share. We need your help to enhance our visibility, so that our platform continues to serve you. #alzheimer #science #health