Rare disease clinical development is evolving rapidly, fueled by regulatory innovation, adaptive trial designs, artificial intelligence, and a more global approach to patient recruitment. Yet success depends on more than scientific advances, it requires operational strategies that address limited ... Rare disease clinical development is evolving rapidly, fueled by regulatory innovation, adaptive trial designs, artificial intelligence, and a more global approach to patient recruitment. Yet success depends on more than scientific advances, it requires operational strategies that address limited patient populations, complex endpoints, and scarce natural history data. She has leveraged her regulatory expertise to support a wide variety of therapeutic treatments (small molecules, antibodies, recombinant proteins, cell and gene therapies, drug/device combination products) designed to treat a broad array of diseases and conditions (hematology, oncology, autoimmune, ophthalmic, cardiovascular, endocrine, pulmonary, CNS, anti-viral, metabolic, dermatology, rare diseases, etc.). Through opportunities working with healthcare organizations on content strategy, writing, speaking, clinical trial development, and sharing the patient experience she aims to affect the healthcare landscape by raising awareness of rare diseases, promoting self-advocacy to patients, and valuable insights to organizations. Jonathan Kornstein is Vice President of Rare Disease and Pediatrics at Caidya, where he leads teams delivering clinical trial services tailored to the unique challenges of rare disease and pediatric development.

Original title: “Practical Strategies: The Future of Rare Disease Clinical Development - Arena International”

in silico analysis of chd4 mutations reveals domain specific impacts on cardiovascular disorders among patients with rare diseases

Original title: “In silico Analysis of CHD4 Mutations Reveals Domain-Specific Impacts on Cardiovascular Disorders Among Patients With Rare Diseases.”