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CLINICAL PERSPECTIVES Phase 2a GATEWAY Trial of PORT-77 in Patients With Erythropoietic Protoporphyria Shows Promise Pete Schmidt, MD, MSc, Chief Medical Officer of Portal Therapeutics, discusses data from the phase 2a GATEWAY trial testingPORT-77, an ABCG2 inhibitor, in patients with erythropoietic protoporphyria (EPP). https://checkrare.com/phase-2a-gateway-trial-of-port-77-in-patients-with-erythropoietic-protoporphyria-shows-promise/ Patient Perspective: Diagnosis and Treatment Journey With ROS1-Positive Non-Small Cell Lung Cancer Jim Herbert, a patient with ROS1-positive non-small cell lung cancer (NSCLC), discusses his diagnosis and treatment journey. https://checkrare.com/patient-perspective-diagnosis-and-treatment-journey-with-ros1-positive-non-small-cell-lung-cancer/ Results from the MajesTEC-9 and CARTITUDE-4 Clinical Trials in Patients With Relapsed/Refractory Multiple Myeloma Roberto Mina, MD, Assistant Professor at Winship Cancer Institute at Emory University, discusses results from the MajesTEC-9 and CARTITUDE-4 clinical trials in patients with relapsed/refractory multiple myeloma (RRMM). https://checkrare.com/results-from-the-majestec-9-and-cartitude-4-clinical-trials-in-patients-with-relapsed-refractory-multiple-myeloma/ Going Beyond Lab Values: Cognitive Impairment in Patients With Immune Thrombocytopenia David Kuter, MD, DPhil, Director of Clinical Hematology at Massachusetts General Hospital and Professor of Medicine at Harvard Medical School, discusses cognitive impairment in patients with immune thrombocytopenia (ITP). https://checkrare.com/going-beyond-lab-values-cognitive-impairment-in-patients-with-immune-thrombocytopenia/ Results From the PROGRESS Study of Zilurgisertib in Patients With Fibrodysplasia Ossificans Progressiva Joanne Quan, MD, Chief Medical Officer of Mirum Pharmaceuticals, discusses results from the PROGRESS study of zilurgisertib in patients with fibrodysplasia ossificans progressiva (FOP). https://checkrare.com/results-from-the-progress-study-of-zilurgisertib-in-patients-with-fibrodysplasia-ossificans-progressiva/ DAHLIAS Clinical Trial Evaluating Nipocalimab in Sjögren’s Disease Robert Hal Scofield, MD, Professor of Medicine, at the University of Oklahoma and Oklahoma Medical Research Foundation, discusses the DAHLIAS clinical trial evaluating nipocalimab in Sjögren’s disease. https://checkrare.com/dahlias-clinical-trial-evaluating-nipocalimab-in-sjogrens-disease/ 2-Year Data on Crinecerfont for the Treatment of Patients With Congenital Adrenal Hyperplasia Oksana Hamidi, DO, Associate Professor at the University of Texas Southwestern Medical Center, discusses 2-year data on crinecerfont for the treatment of patients with congenital adrenal hyperplasia (CAH). https://checkrare.com/2-year-data-on-crinecerfont-for-the-treatment-of-patients-with-congenital-adrenal-hyperplasia/ Lipodystrophy Research Presented at ENDO 2026 Elif Oral, MD, Professor at the University of Michigan, discusses advances in lipodystrophy research presented at ENDO 2026. https://checkrare.com/lipodystrophy-research-presented-at-endo-2026/ Prader-Willi Syndrome: Diazoxide Choline Extended Release Improves Hyperphagia Evelien Gevers, PhD, MD, Consultant Pediatrician and Reader in Endocrinology and Diabetes at Barts Health NHS Trust, discusses effects of diazoxide choline extended release (DCCR) on hyperphagia in patients with Prader-Willi syndrome (PWS). https://checkrare.com/prader-willi-syndrome-diazoxide-choline-extended-release-improves-hyperphagia/ The Importance of Patient and Physician Communication in the Care of Hypoparathyroidism Katie Gillick, Patient Advocate at Orsini Specialty Pharmacy, discusses the importance of patient and physician communication in the care of hypoparathyroidism. https://checkrare.com/the-importance-of-patient-and-physician-communication-in-the-care-of-hypoparathyroidism/ Congenital Adrenal Hyperplasia: Effect of Crenessity (Crinecerfont) on Bone Age Advancement Maria Vogiatzi, MD, Division of Endocrinology at the Children's Hospital of Philadelphia, discusses the effect of Crenessity (crinecerfont) on bone age advancement in patients with congenital adrenal hyperplasia (CAH). https://checkrare.com/congenital-adrenal-hyperplasia-effect-of-crenessity-crinecerfont-on-bone-age-advancement/ Congenital Hyperinsulinism Diagnosis and Management Kristen Rohli, PhD, Associate Director of Research at Congenital Hyperinsulinism International, discusses congenital hyperinsulinism (HI) diagnosis and management. https://checkrare.com/congenital-hyperinsulinism-diagnosis-and-management/ Results from the CALIBRATE Clinical Trial in Patients With Autosomal Dominant Hypocalcemia Type 1 Rachel Gafni, MD, Senior Research Physician at the National Institutes of Health, discusses results from the CALIBRATE clinical trial in patients with autosomal dominant hypocalcemia type 1 (ADH1). https://checkrare.com/results-from-the-calibrate-clinical-trial-in-patients-with-autosomal-dominant-hypocalcemia-type-1/ A Patient’s Diagnostic Journey With Congenital Adrenal Hyperplasia Melanie Gander, mother of a son with congenital adrenal hyperplasia (CAH), and Ambassador with Neurocrine Biosciences, discusses her son’s diagnostic journey with congenital adrenal hyperplasia. https://checkrare.com/a-patients-diagnostic-journey-with-congenital-adrenal-hyperplasia/ Mental Health Challenges and Care Gaps in Patients With Lipodystrophy Kate Stratton, Executive Director of Lipodystrophy United, discusses mental health challenges and care gaps in patients with lipodystrophy. https://checkrare.com/mental-health-challenges-and-care-gaps-in-patients-with-lipodystrophy/ Treatment With Xywav (Low Sodium Oxybate) in Patients With Narcolepsy and Idiopathic Hypersomnia Logan Schneider, MD, Adjunct Clinical Associate Professor of Psychiatry and Behavioral Sciences at Stanford University, discusses treatment with Xywav (low sodium oxybate) in patients with sleep disorders, narcolepsy and idiopathic hypersomnia. https://checkrare.com/treatment-with-xywav-low-sodium-oxybate-in-patients-with-narcolepsy-and-idiopathic-hypersomnia/