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CLINICAL PERSPECTIVES MESA Extension Study of Sevasemten in Patients With Becker Muscular Dystrophy https://checkrare.com/mesa-extension-study-of-sevasemten-in-patients-with-becker-muscular-dystrophy/ Joanne Donavan, MD, PhD, Chief Medical Officer at Edgewise Therapeutics, discusses the MESA extension study of sevasemten for the treatment of patients with Becker muscular dystrophy (BMD). 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 Cureus analyzed a case study of an adolescent with Rapid Eye Movement (REM) sleep behaviour disorder in Moebius syndrome. How The FDA’s Plausible Mechanism Framework Is Accelerating Approval for Osteosarcoma Immunotherapy https://checkrare.com/how-the-fdas-plausible-mechanism-framework-is-accelerating-approval-for-osteosarcoma-immunotherapy/ Paul Romness, CEO of OS Therapies, discusses the Plausible Mechanism Framework draft guidance and how it will affect the approval process of OST-HER2 LM for the treatment of osteosarcoma. CHMP Recommends Mavorixafor for WHIM Syndrome https://checkrare.com/chmp-recommends-mavorixafor-for-whim-syndrome/ The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion recommending marketing authorization for mavorixafor (Xolremdi) for the treatment of patients with WHIM syndrome. CureSHANK: Update on Clinical Research in Phelan-McDermid Syndrome https://checkrare.com/cureshank-update-on-clinical-research-in-phelan-mcdermid-syndrome/ Geraldine Bliss, Co-Founder and President of CureSHANK, and Jenny Graham Beeson, Board Member for CureSHANK, discuss clinical research in Phelan-McDermid syndrome (PMS) and the Start Genetic initiative. New Clinical Trial Testing On-Demand Use of Deucrictibant in Patients With Hereditary Angioedema https://checkrare.com/new-clinical-trial-testing-on-demand-use-of-deucrictibant-in-patients-with-hereditary-angioedema/ Peng Lu, MD, PhD, Chief Medical Officer at Pharvaris, and Wim Souverijns, PhD, Chief Commercial Officer at Pharvaris, discuss the RAPIDe-3 clinical trial of deucrictibant for patients with hereditary angioedema (HAE). First Patient Dosed in FALCON Clinical Trial Evaluating SGT-212 Gene Therapy for Friedreich’s Ataxia https://checkrare.com/first-patient-dosed-in-falcon-clinical-trial-evaluating-sgt-212-gene-therapy-for-friedreichs-ataxia/ Gabriel Brooks, MD, Chief Medical Officer at Solid Biosciences, and Russell Lonser, MD, of The Ohio State University Wexner Medical Center, discuss SGT-212 and the FALCON clinical trial for patients with Friedreich's ataxia. Gene Therapy in Patients With MPS IIIA https://checkrare.com/gene-therapy-in-patients-with-mps-iiia/ Brian Bigger, PhD, Professor of Advanced Therapeutics at the University of Edinburgh, discusses hematopoietic stem cell gene therapy (HSCGT) in patients with mucopolysaccharidosis IIIA (MPS IIIA; Sanfilippo syndrome type A). PRIZM Clinical Trial of Zagociguat in Patients With MELAS https://checkrare.com/prizm-clinical-trial-of-zagociguat-in-patients-with-melas/ Amel Karaa, MD, Genetics and General Metabolism, Director of the Mitochondrial Disease Program at Massachusetts General Hospital, discusses the PRIZM clinical trial of zagociguat in patients with MELAS. Chiesi Rare Disease Highlights at WORLDSymposium 2026 https://checkrare.com/chiesi-rare-disease-highlights-at-worldsymposia-2026/ Rachele Berria, MD, PhD, Senior Vice President, Head of Global Medical Affairs at Chiesi Rare Diseases, gives an overview of the company’s highlights at WORLDSymposia 2026. Real-World Perspective on Daily Challenges of Living With MPS II https://checkrare.com/real-world-perspective-on-daily-challenges-of-living-with-mps-ii/ Kristin McKay, President and Executive Director of Project Alive, discusses the real-world perspective on daily challenges of living with mucopolysaccharides II (MPS II).