CobiCure

Rare Disease Day, observed annually on the last day of February, shines a light on the challenges faced by the 300 million people worldwide living with rare diseases. Held on February 28 (or February 29 during Leap Years—the rarest day on the calendar), this day raises awareness of rare diseases as a global public health concern. Originally launched in Europe in 2008 by EURORDIS, Rare Disease Day expanded to the U.S. in 2009 in partnership with NORD (National Organization for Rare Disorders). Today, it’s celebrated in over 85 countries, bringing together patients, families, researchers, advocates, and communities to share stories, host events, and educate others about the realities of living with rare diseases.  How can you participate? 💙 Share a story, blog, or video to amplify the voices of those affected. 🧡 Join a virtual or in-person event near you. 🩵 Learn more about rare diseases and how to advocate by visiting rarediseaseday.us or rarediseaseday.org. Together, we can raise awareness, support innovation, and create a brighter future for those impacted by rare diseases. 

Healthcare innovation is failing the most vulnerable patients – children who have serious medical conditions, with market sizes too small to justify industry investment. CobiCure aims to change that equation by collaborating with others who are passionate about finding solutions. We bring together clinical, biopharma, and medical technology innovators to overcome the barriers that prevent the delivery of new life-saving drugs and devices to the sick children who need them. Discover the mission of CobiCure – and learn how we’re breaking down barriers and working to saving kids’ lives. Read Michele’s article here: https://lnkd.in/gqmNeFjj

Rare diseases impact millions, yet their challenges are staggering—especially for children. 30% of children with rare diseases pass away before their fifth birthday, due to factors like delayed diagnoses, limited treatment options (95% of rare diseases lack approved treatments), and the complex, lifelong nature of these conditions. Research hurdles, insufficient medical training, and low public awareness further exacerbate the problem, leaving families with few paths forward. At CobiCure, we are dedicated to changing this narrative. By driving the development of innovative pediatric medical technologies, we aim to address these barriers and bring life-changing possibilities to children and families impacted by rare diseases.

Dr. Stefano Pezzato brings a wealth of expertise in anesthesia, intensive care, and pediatric science. With a Ph.D. in Pediatric Science, he spent eight years at IRCCS Istituto Gaslini in Genova, Italy, managing complex cases, including cardiac surgery and ECMO. His research, shaped by international collaborations, focuses on advanced monitoring in pediatric neurocritical care. In 2022, he joined Children's National Hospital in Washington, D.C., as a Research Postdoctoral Fellow, where he develops multimodal neuromonitoring for infants and children undergoing open-heart surgery. Dr. Pezzato’s mission is to bridge the gap between research and daily clinical practice. He values the opportunity to work on diverse projects, gaining insights into the device innovation process across multiple phases and leveraging his multidisciplinary experience. One of the highlights of his work is engaging with experts during weekly lectures inspired by the Stanford Biodesign program. These interactions, enriched by diverse MedTech expertise, provide invaluable perspectives on advancing pediatric care.

Rare diseases may seem uncommon, but collectively, they impact an estimated 25 to 30 million Americans—that’s 1 in 10 people. To put it in perspective, that’s one person on every elevator and four on every bus. With over 7,000 rare diseases identified by the NIH, they span the entire medical spectrum, from neurological and metabolic conditions to chromosomal disorders and rare cancers. While some, like cystic fibrosis, are widely recognized, others, such as cat eye syndrome, remain less known. Many rare diseases bear the names of the physicians, patients, or even hospitals that first identified them. Rare diseases affect people of all ages, with approximately two-thirds of those impacted being children. Whether genetic, visible at birth, or appearing later in life, these conditions present unique challenges for patients, families, and the healthcare system. At CobiCure, we recognize that rare diseases are more than a medical challenge—they are a profound public health concern. Our mission is to enable the development of pioneering pediatric medical technologies and therapeutics, ensuring children and families facing rare diseases have access to innovative solutions that transform lives.

We’ve reached the end of our series recounting the amazing day we had hosting our Pediatric MedTech CEOs in November, and this final takeaway is one that many of us really need to be keyed into. Kolaleh Eskandanian led panelists Kunj Sheth, Andre Bessette, and Jo CarolHiatt in a highly informative discussion on hospital & payer contracting, providing an excellent overview of the timelines and procedures of value analysis committees (VAC). It was noted that clinical evidence is a primary driver, but it isn’t everything. For example, devices that replace something else are typically more favorable than devices that are adding to the overall system without taking anything away. Impact1 at Stanford Biodesign CobiCure has SO many exciting things coming up to share with you: from our MedTech Fellowship to the introduction of new initiatives, you’ll want to stay connected to our community to be part of the progress!

Dr. Tamara Lambert is a CobiCure Fellow working with Dr. Juan Espinoza to develop a pediatric medical device for early sepsis detection in oncology patients. She holds degrees in Biological Engineering, Public Health, Bioengineering, and a Ph.D. in Biomedical Engineering. Her work has earned recognition in the startup space, including as a finalist in the NIA Startup Challenge and a Wearable Tech Ventures Ambassador. Dr. Lambert’s passion is using innovation to address healthcare disparities in underserved populations. Her drive comes from personal experiences and witnessing inequities in countries like Nicaragua and India. She is committed to developing affordable, accessible technologies to improve care for marginalized communities. Her mission? To tackle healthcare disparities among the youngest and sickest patients, creating technologies that lead to meaningful improvements in their lives. Dr. Lambert values collaborating with passionate innovators and gaining insights into barriers to pediatric innovation, further fueling her dedication to advancing equitable healthcare solutions. Dr. Lambert is dedicated to turning her vision into impactful technologies that bring better healthcare to those who need it most.

Tomorrow is International Childhood Cancer Day. We stand united with children and adolescents battling cancer, as well as survivors and their families. This global campaign serves as a powerful reminder of the urgent need for equitable access to life-saving treatments and care for every child diagnosed with cancer. At Cobicure, we're committed to being part of the solution. We are actively connecting with potential partners, collaborators, and physicians to explore how we can develop better treatments for these brave children and give them hope for the future. 💛🎗️

During our CEO Summit, Eric Chehab and Kevin Unger joined CobiCure Fellow Juliana Perl to walk our Pediatric MedTech CEO Summit through go to market strategies. They noted that sales and distribution channels will be driven by what is available to you as a company at the time, but will always have the potential to evolve over time as you grow and expand. In fact, many companies have had lots of success investing in clinical education and making sure this is a primary focus of their commercialization strategies. Impact1 at Stanford Biodesign

Rare diseases touch the lives of many, often in unexpected ways. While many rare diseases are genetic, they can present at birth or emerge later in life. According to the National Organization for Rare Disorders (NORD), approximately two-thirds of Americans living with rare diseases are children, making this a critical public health issue that affects us all—directly or indirectly. We are all connected to someone navigating a rare disease journey. Let’s work together to raise awareness, advance care, and support those impacted by these unique challenges.