Michael Thurn’s Post

The FDA's announcement of the new Rare Disease Innovation Hub (https://lnkd.in/gPNBec7V) is a significant step forward for the rare disease community. This initiative not only represents progress for regulatory practices but also promises to accelerate the development of much-needed treatments for patients with rare diseases. As a company deeply invested in the biotech and biopharma industries, particularly in the rare disease space, we're so excited about this hub's potential impact. The challenges faced by companies like Stealth BioTherapeutics, who have received conflicting guidance from different FDA divisions, highlight the need for a more cohesive approach. The new hub promises to address these inconsistencies and promote the development of innovative trial designs, novel endpoints, and advanced biomarker tests. At BlueGene Search, we understand the critical importance of supporting advancements leading to more effective and timely therapies. We look forward to seeing the positive outcomes this hub will bring to the rare disease community. We'd love to hear your thoughts on the new Rare Disease Innovation Hub - chime in below! #RareDisease #FDA #Biotech #Innovation #BlueGeneSearch #Biopharma #RareDiseaseHub #RegulatoryAdvancements #HealthcareInnovation

Patient finding in rare disease includes the set of strategies and tactics to find and diagnose patients living with rare or specialty conditions. It sounds straightforward, but that's not always the case. When it comes to developing and commercializing associated treatments, this piece of the puzzle is critical not only for biopharma companies in their journey to help patients living with life-altering conditions, but the patients who are seeking answers. Explore our approach to patient finding: https://hubs.li/Q02Lz9SW0 #patientfinding #patientfinding101 #raredisease #specialtydisease #rarediseasediagnosis #biopharma #biotech

Doesn't everyone want to slow down the aging process and minimize or eliminate the associated illness with aging? The announcement from Rubedo Life Sciences marks a significant milestone in the fight against age-related disease. The largest segment of the population that will require the most healthcare in history will be our elderly population over the next 20 years. With $40 million in financial backing, the company is poised to initiate human trials for its groundbreaking drug, RLS-1469, which targets senescent cells—a key driver of age-related illnesses. This development holds promise for addressing a wide range of age-related conditions, from cardiovascular disease to neurodegenerative disorders. By specifically targeting senescent cells, RLS-1469 has the potential to not only alleviate symptoms but also halt or even reverse the progression of these debilitating diseases. This announcement represents a significant step forward in the field of biopharmaceuticals and offers hope for a future where age-related diseases can be effectively treated, minimized, delayed or even prevented. More info on the announcement here: https://lnkd.in/e5Kkk_HK #AntiAging #ClinicalTrials #Biopharma #LifeSciences #Biotech #Medicine

Did you know that #glaucoma is the leading cause of irreversible blindness worldwide? Glaucoma is a disease of the optic nerve which, if left untreated, can lead to irreversible vision loss. It affects about 3.5% of the global population, yet current treatment options can still lead to disease progression in many patients. Glaucoma is also largely underdiagnosed, including in Western countries where less than 50% of people affected are actually treated, which leaves an opportunity to improve the standard of care. Currently, reducing elevated intraocular pressure (IOP) remains the only way to slow progression of the disease. But, as many as 40% of patients fail to reach target IOP with existing monotherapies, risking disease progression and permanent vision loss. There continues to be an unmet medical need for a therapy with greater IOP-lowering efficacy that is both safe and well tolerated, like Nicox’s Phase 3 investigational drug NCX 470, despite that we have established first line therapies. #blindness #eyedisease #healthcare #biotech

Last Tuesday our CMO John Wagner, MD, PhD joined jp clancy, Andrew E. M., Robert Schuck and Nancy M. Allen LaPointe in a lively discussion around Integrating Sensitive Endpoints in Early-Phase Trials at Duke-Margolis Institute for Health Policy’s workshop around Opportunities to Improve Dose-Finding and Optimization for Rare Disease Drug Development. The discussion included best practices and considerations when selecting appropriate clinical outcome assessments that are also meaningful to patients, with a focus on strategies for integrating sensitive endpoints in early phase trials, including digital measurements. #drugdevelopment #digitalbiomarkers #biomarkers

What an interesting read on the rise of patient-led clinical research in rare diseases! Check out our latest blog to learn about the latest shift in rare clinical trials and how patient organizations have helped advance drug development efforts: https://bit.ly/48Q3tZY

What an interesting read on the rise of patient-led clinical research in rare diseases! Check out our latest blog to learn about the latest shift in rare clinical trials and how patient organizations have helped advance drug development efforts: https://bit.ly/3Odedbv

What an interesting read on the rise of patient-led clinical research in rare diseases! Check out our latest blog to learn about the latest shift in rare clinical trials and how patient organizations have helped advance drug development efforts: https://bit.ly/4hEVjrh

Clinical trial participants often require regular monitoring to assess drug efficacy, but frequent blood draws can reduce compliance and increase participant discomfort. Invasive testing can discourage participants from staying in clinical trials, potentially compromising the quality and quantity of the data collected. Metabolic Solutions’ breath tests are non-invasive and easily repeatable, making them ideal for clinical trials requiring regular, frequent assessments.   Our tests provide high-quality data without causing discomfort, helping ensure participant retention and data integrity. Learn more at https://lnkd.in/er_X2ub6 #LiverHealth #LiverDiseaseResearch #NASH #ClinicalTrials #LiverCancer #LiverCare #PharmaResearch

What an interesting read on the rise of patient-led clinical research in rare diseases! Check out our latest blog to learn about the latest shift in rare clinical trials and how patient organizations have helped advance drug development efforts: https://bit.ly/41hWzv2