Sano Genetics’ Post

It is possible for drug discovery and development of new molecules to happen with a collaborative persuasion. The pre-competitive space is actually a public domain that holds great benefit when developing disease pathways. These can be really long or surprisingly short but nonetheless control our patient's response to the world of injury, infection, stress, hunger and fed states. Even more fascinating is what is possible in determining the markers of disease. Interestingly, this shared space that begins with none of the consortium of players seeking individual gain, leads to prognostic or diagnostic indicators that could ultimately help us conduct stratification of patient populations. Here is why this is important, it fuels the pursuit of personalized medicine that answers the question "With my patient's profile, are they likely to benefit from this intervention or should i pursue another?" This gets me especially excited because #CompoundingPharmacy is one of the best expressions of personalized, precision medicine! #IndustrialPharmacy #PrecisionMedicine #PersonalizedMedicine #RegulatoryAffairs #CompoundingSpecialist

A patient-led organization has just made an incredible breakthrough by repurposing a drug for a rare and deadly bleeding condition! Recently, a large clinical trial revealed that pomalidomide, originally developed for multiple myeloma, has proven effective in treating a life-threatening bleeding disorder called HHT (Hereditary Hemorrhagic Telangiectasia). This drug not only reduced bleeding events but also improved patients’ quality of life. Cure HHT brought together physicians, researchers, and patients to push this repurposing opportunity forward, showing the true power of patient-led organizations. At Every Cure, we aim to work closely with patient organizations to identify the most promising opportunities from our platform and ensure the necessary trials are conducted to benefit as many patients as possible. Because no one understands a rare disease better than those living with it. #raredisease #drugdevelopment #drugrepurposing #clinicaltrial

I was honored to deliver a keynote address at the World Orphan Drug Congress USA in Boston yesterday, focused on the opportunity to drive innovation and increase treatment options in rare diseases. Innovation in clinical trial design, evidence generation and evaluation are necessary steps in improving access to therapy for people living with rare diseases. But first, there needs to be alignment across stakeholder groups. If we can collectively agree on the use of evidence generation and interpretation methods, then we can better address the complex challenges in rare diseases, advance new medicines for patients, and ultimately bring better care for rare. #raredisease #orphandrug #WorldOrphanUSA

The success of your drug development relies on access to proven Parkinson’s disease expertise and robust processes and technologies to enhance your trial design and accelerate development. Clario's globally recognized scientific and medical experts provide the evidence you need to address challenges such as: ▫️ Early disease detection for timely treatment intervention with investigational treatments ▫️ Accounting for significant person-to-person differences in your study design ▫️ Strategies for accelerating timelines ▫️ Optimizing your clinical outcome measures ➡️ Watch our webinar to learn how Clario can help you navigate your Parkinson's trial more effectively. https://lnkd.in/d9ts99XD #ClinicalTrials #ParkinsonsDisease

The success of drug development in Parkinson’s disease lies in its ability to enhance patients’ quality of life through effective symptom management. However, the real breakthrough will come when therapies can slow or stop disease progression. As our understanding of the disease’s underlying mechanisms deepens, particularly regarding α-synuclein and neuroinflammation, we are on the brink of developing transformative treatments. The focus now should be on precision medicine, early diagnosis, and neuroprotective strategies to truly redefine outcomes for Parkinson’s patients. Congratulations to Clario for its leadership in advancing clinical research solutions! Their commitment to innovation and technology-driven services is transforming the way clinical trials are conducted, making significant contributions to patient-centric research and drug development. Their success highlights the importance of integrating digital solutions to enhance data accuracy, streamline processes, and ultimately improve patient outcomes.

Join Clario on May 16 for an enlightening webinar that explores Parkinson’s disease endpoint data collection. Our experts will delve into topics that include: -Sensitivity of digital measures in disease progression monitoring -Efficacy and quality of life evaluations using clinical scales and patient-reported outcomes -Imaging features for biomarker-guided drug development Register now to gain invaluable insights in Parkinson's disease research: https://lnkd.in/eCN3y5jb #ParkinsonsResearch #EndpointDataCollection #Webinar

New data to help in treatment decisions for Crohn's disease patients! A study recently published in the New England Journal of Medicine shows that risankizumab, brand name SKYRIZI® is more effective than ustekinumab, brand name STELARA® in achieving clinical and endoscopic remission in patients with moderate-to-severe Crohn’s disease who had unacceptable side effects with anti-TNF therapy or an inadequate response to such therapy. Significant strides in treatment options! Find the clinical evidence for both #SKYRIZI & #STELARA on DRUGDOCS® Read the paper here: https://lnkd.in/ex5znVzp #CrohnsDisease #MedicalResearch #IBD

Important analysis from Simone Villa, Pierpaolo de Colombani, Lucia Dall’Olio, Giuliano Gargioni, and Mario Raviglione presenting crucial #policy recommendations aimed at enhancing clinical trials for new #tuberculosis drug regimens. This comprehensive approach could significantly impact TB treatment and control efforts globally. 👉 Read the full paper for in-depth insights and implications: https://lnkd.in/ekHiNyRg #TBResearch #ClinicalTrials #GlobalHealth UNITE4TB Innovative Health Initiative (IHI)

Needle in a haystack? Try finding patients for a rare disease trial! Whenever I plan a clinical trial in a rare disease, I always start by asking two questions: ➡ Where are the patients? ➡ Can you identify the patients? These questions are at the core of any successful trial in this space. How much it will cost or how long it will take—comes secondary. What questions do you ask first? #ClinicalTrials #RareDiseases #DrugDevelopment #Biotech #ClinicalOperations #Strategy

Are we entering a new era for the treatment of (at least a subtype) of #parkinsondisease. There is room for optimism, as well as caution till we reasonably grasp the effects of GLP-1 agonists on pathogenic processes driving neuronal dysfunction in PD. For a long time, there was a dogma on peptic ulcer disease that it is caused by excessive acid in stomach. Now, we know that the condition can be treated in vast majority by antibiotics to eradicate H. pylori. I am afraid that we face similar dogmas in PD research (ie. role of excessive synuclein accumulation thought as the cause). We shared our reflections on this topic https://lnkd.in/dEgNDtdd