CLINICAL PERSPECTIVE (+++ +++ +++)

CLINICAL PERSPECTIVE Data on Maribavir in Post-Transplant Cytomegalovirus Neil Inhaber, MD, FRCPC, Therapeutic Area Head of Global Medical, Rare Disease at Takeda, discusses data on maribavir in post-transplant cytomegalovirus (CMV) presented at the American Transplant Congress 2026. https://checkrare.com/data-on-maribavir-in-post-transplant-cytomegalovirus/ New Graves’ Disease Community Website Christine Gustafson, Executive Director and CEO of the TED Community Organization, discusses the new Graves’ Community website. https://checkrare.com/new-graves-disease-community-website/ Narcolepsy Diagnosis and Symptoms: Understanding the Daily Burden of the Disease Tammy Anderson, Executive Director, and Jenny Rose, Manager of Outreach and Management at Wake Up Narcolepsy, discuss the challenges of diagnosing narcolepsy and the profound impact the disorder can have on patients and their families. Both Ms. Anderson and Ms. Rose are parents of children with narcolepsy. https://checkrare.com/narcolepsy-diagnosis-and-symptoms-understanding-the-daily-burden-of-the-disease/ Kleefstra Syndrome: Development of RNA Amplifier Therapy Eric Scheeff, PhD, Chief Scientific Officer at iDefine, discusses the treatment development of RNA amplifiers in patients with Kleefstra syndrome. https://checkrare.com/kleefstra-syndrome-development-of-rna-amplifier-therapy/ Predicting Treatment Response Outcomes With Proteomic and Machine Learning Analyses in Myasthenia Gravis Henry Kaminski, MD, Department of Neurology, and Linda Kusner, PhD, Department of Pharmacology & Physiology at George Washington University, discuss predicting response outcomes with proteomic and machine learning analyses in treating patients with myasthenia gravis (MG). https://checkrare.com/predicting-treatment-response-outcomes-with-proteomic-and-machine-learning-analyses-in-myasthenia-gravis/ Making Travel Accessible: SMAshing My Limits Partners With Becoming rentABLE Tracey Dawson, PhD, Senior Vice President, US Therapeutic Area Head Neuroscience at Novartis, discusses the collaboration between Novartis, SMAshing My Limits, and Becoming rentABLE, striving to make travel accessible. https://checkrare.com/making-travel-accessible-smashing-my-limits-partners-with-becoming-rentable/ Addressing Patient Experiences With IgA Nephropathy Gaia Coppock, MD, Nephrologist and Associate Professor of Clinical Medicine at the University of Pennsylvania, discusses patient experiences with IgA nephropathy (IgAN). https://checkrare.com/addressing-patient-experiences-with-iga-nephropathy/ Patient Perspective: Diagnostic Journey With IgA Nephropathy Byron Wade, a patient with IgA nephropathy (IgAN), discusses his personal diagnostic journey and a recent study looking at other patients’ experiences. https://checkrare.com/patient-perspective-diagnostic-journey-with-iga-nephropathy/ Importance of Patient Listening in The Development of Rare Disease Treatments Matt Trudeau, President of ITF Therapeutics, discusses the importance of patient listening in the development of Duvyzat (givinostat) for Duchenne muscular dystrophy (DMD). https://checkrare.com/importance-of-patient-listening-in-the-development-of-rare-disease-treatments/