CLINICAL PERSPECTIVES +++ +++ +++ (videos)

CLINICAL PERSPECTIVES 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. Nipocalimab Granted Fast Track Designation in Systemic Lupus Erythematosus https://checkrare.com/nipocalimab-granted-fast-track-designation-in-systemic-lupus-erythematosus/ Richard A. Furie, MD, Chief of the Division of Rheumatology at Northwell Health, discusses nipocalimab for the treatment of systemic lupus erythematosus (SLE). 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 WHIM syndrome in patients aged 12 years and older. CureSHANK: Update on Clinical Research in Phelan-McDermid Syndrome by Madaline Spencer| Published on: Mar 23, 2026 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). Challenges and Unmet Needs of Female Patients With Fabry Disease https://checkrare.com/challenges-and-unmet-needs-of-female-patients-with-fabry-disease/ Dawn Laney, MS, Genetic Counselor at Emory University School of Medicine, discusses the challenges and unmet needs of female patients with Fabry disease. Global Efforts Towards Developing Treatment Options for Patients With Rare Lysosomal Storage Disorders https://checkrare.com/global-efforts-towards-developing-treatment-options-for-patients-with-rare-lysosomal-storage-disorders/ Bob Stevens, Group CEO of the MPS Society and Rare Disease Research Partners in the UK, discusses global efforts towards developing treatment options for patients with rare lysosomal storage disorders (LSDs). Positive Topline Results from a Study Testing Nipocalimab in Patients With Systemic Lupus Erythematosus https://checkrare.com/positive-topline-results-from-a-study-testing-nipocalimab-in-patients-with-systemic-lupus-erythematosus/ Leonard L. Dragone, MD, PhD, Disease Area Leader in Autoantibody and Rheumatology, Johnson & Johnson Innovative Medicine, discusses positive topline results from a study of nipocalimab in patients with systemic lupus erythematosus (SLE). New Formulation of Berotralstat for Treatment of Pediatric Patients With Hereditary Angioedema https://checkrare.com/new-formulation-of-berotralstat-for-treatment-of-pediatric-patients-with-hereditary-angioedema/ Raffi Tachdjian, MD, Associate Clinical Professor of Medicine & Pediatrics, Division of Allergy & Clinical Immunology, University of California Los Angeles, discusses a new formulation of Orladeyo (berotralstat) for the treatment of pediatric patients with hereditary angioedema (HAE).