AlloVir的动态

The GARDIAN Registry has released data on the first 26 patients: 96% with Gaucher disease Type 3 (GD3) and 4% with Type 2 (GD2). GARDIAN (Gaucher Registry for Development, Innovation, and Analysis of Neuronopathic Disease) is a global initiative by the International Gaucher Alliance (IGA). It aims to improve understanding of GD2 and GD3 by collecting personal experiences from patients and families. At Prevail Therapeutics, we celebrate the remarkable work of the IGA as we recognize the critical need to include patient and caregiver voices throughout the drug development process. By collaborating with the IGA to help support GARDIAN, we hope to develop a more authentic understanding of the patient experience that will improve current drug development efforts. Discover more about the results here: https://bit.ly/4eipC5w #GARDIANRegistry #GaucherDisease #InternationalGaucherAlliance #PatientDrivenResearch #InnovationInHealthcare

Studies show that when it comes to liver disease treatment, Proton Density Fat Fraction is the best predictor of response. Livivos's proprietary non-invasive technology promises to deliver the accuracy of MRI PDFF in point-of-care settings. Follow our journey www.livivos.com Livivos, Inc #LiverScope #MASH #NASH #patientcentric #pointofcare #liverdisease #fattyliver #PDFF #NAFLD #rezdiffra #clinicaltrials #resmetirom Madrigal Pharmaceuticals Mazen Noureddin, MD, MHSc LiverScope is an investigational device. Limited by Federal (or United States) law to investigational use.

?? ?????? ???????? ???? ?????? ???????? ?????????????? ??????????! ?? Immedica Pharma AB has announced its agreement to acquire Marinus Pharma, marking a transformative milestone in the fight against rare seizure disorders. This acquisition brings ZTALMY? (ganaxolone) oral suspension, an FDA-approved breakthrough for treating CDKL5 deficiency disorder (CDD) seizures, to Immedica's portfolio. This move expands Immedica's rare disease focus into North America and strengthens its mission to deliver life-changing treatments for underserved patients. ?? What does this mean for patients? More access to innovative therapies and hope for improved outcomes. ?? ?????????? ???????? ?????????? ???????? ?????????????????? ?????????????????????????? ????????: https://lnkd.in/gdY7HyFm #rarediseases #pharmainnovation #seizuredisorders #pharmaceuticalnews #healthcareinnovation #immedicapharma #marinuspharma #medicalbreakthroughs #fdaapproved #rarediseasetreatment #globalhealth #healthcareleadership

This week, MAIA announced that the FDA has designated THIO for the treatment of pediatric-type diffuse high-grade gliomas (#PDHGG) as a drug for a “rare pediatric disease.” MAIA’s Vice President and Head of Regulatory and Quality K. Robinson Lewis comments, “Rare pediatric disease designation also offers a highly valuable incentive for MAIA. Upon FDA approval of a future new drug application in PDHGG, MAIA would be eligible to receive a priority review voucher that can be redeemed or sold as an asset at a very high valuation.” Previous research showcased THIO’s potency as a treatment for a PDHGG subtype known as diffuse intrinsic pontine glioma (DIPG). In case you missed the news, dive into the press release here: https://bit.ly/3ZHubzQ #Biotech

Eli Lilly and Company wins head-to-head weight loss trial against Novo Nordisk The market for first-generation weight loss treatments has been dominated by two major players, Eli Lilly and Novo Nordisk. But new data provide the clearest evidence yet that one may have an edge. On Wednesday, Lilly reported the first data from a head-to-head Phase 3 trial testing Zepbound against Novo’s Wegovy, finding that Zepbound was the superior weight loss agent. By week 72, patients given Zepbound lost 20.2% of their body weight on average, compared to 13.7% for Wegovy, the study’s primary endpoint. That equated to a 47% greater relative weight reduction, Lilly said. The percentage changes in weight translated to #Zepbound patients losing an average of 50.3 pounds. For patients given #Wegovy, the average weight loss was 33.1 pounds. https://lnkd.in/gDCQ2Aqz

Missed our keynote session “Can you overcome complexity and deliver value for rare disease patients in Europe?" Review the summary

The news of this setback is devastating for our Sanfilippo Community. My colleague Matthew Ellinwood has been very open with the facts. I can't comprehend why a treatment with such significant benefits for a community can fail to make it through the development process. I understand the economic and business aspects of this issue. Still, I find it perplexing that solid scientific advancements for ultra-rare diseases face challenges similar to those for more common diseases when seeking approval from the FDA. Allievex had worked closely with the key stakeholders to establish confidence in an Accelerated Pathway with Biomarker considerations. Yet, this product for our Sanfilippo B community will no longer be available. What does this mean? It means that children who have seen improvements from this treatment through the clinical study will lose access to the drug once it runs out in the coming months. Children will start to deteriorate and ultimately lose their battle. This cannot be allowed to happen in the same way for families and their loved ones. Focus on ethical considerations for patients in a study and can we collaborate and create guidelines for exit strategies that won't harm those participating in a clinical study? Picture a foundation that could support a successful therapy and provide the drug to a patient if the clinical research falls through. Surely, this is feasible. Indeed, we need leaders who prioritize the well-being of the patients and who think ambitiously and boldly. My heart aches. And my frustration is mounting.

Today, we announced the U.S. FDA has cleared our Investigational New Drug Application for CLN-978 in systemic lupus erythematosus (#SLE). ?CLN-978 is the first development stage CD19 T cell engager to receive IND Clearance in #autoimmune diseases. CLN-978 is a novel bispecific T cell engager that targets CD19, offering a highly differentiated approach to deliver the potency of T cell redirecting therapy with off-the-shelf access and convenient dosing through subcutaneous administration.? We are advancing CLN-978 in our global Phase 1 clinical trial for patients living with SLE.?? Learn more: https://bit.ly/3zX2F8P

? 2024: A Breakthrough Year in Liver Disease Therapies ? This year brought remarkable advancements in addressing high unmet medical needs, especially in liver disease. ?? FDA approvals spotlighted: ? The first-ever NASH treatment, Madrigal’s Rezdiffra (resmetirom), launched after decades of R&D hurdles, achieving $76.8M in its first months. ? Ipsen/Genfit’s Iqirvo (elafibranor) and Gilead’s Livdelzi (seladelpar) introduced safer, more effective options for PBC, expanding the treatment landscape. Experts agree: while innovation is soaring, success will depend on streamlining care and building disease awareness to ensure these breakthroughs reach patients. ?? From Merck’s Winrevair to Novartis’s Fabhalta, this year proved that the path from lab to life-changing impact is more than approvals—it’s about creating access and momentum. What are your thoughts on the future of these therapeutic areas? Let’s discuss! ?? #PharmaInnovation #LiverDisease #DrugDevelopment #NASH #PBC #HealthcareBreakthroughs #Mash #Citeline #Merck #Madrigal #Gilead #Novartis #Lilly

Hi all, Please consider signing this petition to urge NYS to introduce RiVive, a 3 mg formulation of intranasal naloxone. There is no evidence to support high dose naloxone and we know that high naloxone precipitates significantly more acute opioid withdrawal in comparison with 4 mg or 3 mg IN naloxone. Harm Reduction Therapeutics, the company that manufactures RiVive, is a 501c3 that brought RiVive, the 3mg nasal naloxone, to the market. As a non-profit pharmaceutical company, HRT wants to ensure that people who need naloxone can get it at an affordable price with no mark-up. HRT has partnered with Remedy Alliance to whom they give 10% of all of the RiVive profits to distribute to unfunded programs with no access or budget for naloxone.? Note: I do not work for HRT nor Remedy Alliance and derive no personal benefit from endorsing RiVive. I just support compassionate overdose responses. Link to the petition: https://chng.it/jLc9Km8PYS Thanks, friends.