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

CLINICAL PERSPECTIVE Investigational Dual AAV Gene Therapy for Patients With Stargardt Disease https://checkrare.com/investigational-dual-aav-gene-therapy-for-patients-with-stargardt-disease/ Miquel Vila-Perello, PhD, co-founder and CEO of SpliceBio, discusses an investigational dual adeno-associated virus (AAV) gene therapy in development for patients with Stargardt disease. Psychological Aspects of Rare Autoimmune and Ophthalmic Diseases https://checkrare.com/psychological-aspects-of-rare-autoimmune-and-ophthalmic-diseases/ A study published in World Journal of Clinical Cases investigated the psychological aspects of rare autoimmune and ophthalmic diseases, such as mental well-being and everyday functioning. Rapid Eye Movement Sleep Behaviour Disorder in Moebius Syndrome https://checkrare.com/rapid-eye-movement-sleep-behaviour-disorder-in-moebius-syndrome/ A study published in The Cureus Journal of Medical Science analyzed a case study of an adolescent with Rapid Eye Movement (REM) sleep behaviour disorder in Moebius syndrome. FDA’s Plausible Mechanism Framework and its Effect on Rare Disease Therapy Development https://checkrare.com/fdas-plausible-mechanism-framework-and-its-effect-on-rare-disease-therapy-development/ Stevie Ringel, CEO of Nome Therapeutics, discusses the US Food and Drug Administration’s (FDA) Plausible Mechanism Framework and its effect on rare disease therapy development. Phase 2 Results of Dusquetide in the Treatment of Oral Ulcers Caused by Behçet’s Disease https://checkrare.com/phase-2-results-of-dusquetide-in-the-treatment-of-oral-ulcers-caused-by-behcets-disease/ Results from a phase 2a study evaluating SGX945 (dusquetide) for the treatment of Behçet’s disease were published in Rheumatology (Oxford). Advancing Thyroid Eye Disease Care: Integrating Tepezza, New Technology, and Optimizing Multidisciplinary Teams https://checkrare.com/advancing-thyroid-eye-disease-care-integrating-tepezza-new-technology-and-optimizing-multidisciplinary-teams/ Maja Kostic, MD, PhD, Assistant Professor of Ophthalmology at the University of North Carolina at Chapel Hill, discusses advancements in thyroid eye disease (TED) care. Results from the TEASE-2 Clinical Trial of Gildeuretinol in Patients With Stargardt Disease https://checkrare.com/results-from-the-tease-2-clinical-trial-of-gildeuretinol-in-patients-with-stargardt-disease/ Philip J. Ferrone, MD, Vitreoretinal Consultants of New York, discusses results from the TEASE-2 clinical trial of gildeuretinol in patients with Stargardt disease.