CLINICAL PERSPECTIVE +++++

CLINICAL PERSPECTIVE Positive Topline Results from the CALIBRATE Trial of Encaleret in Patients With Autosomal Dominant Hypocalcemia Type 1 https://checkrare.com/positive-topline-results-from-the-calibrate-trial-of-encaleret-in-patients-with-autosomal-dominant-hypocalcemia-type-1/ Michael A. Levine, MD, ML, Children’s Hospital of Philadelphia, discusses positive topline results from the CALIBRATE trial of encaleret in patients with autosomal dominant hypocalcemia type 1 (ADH1). A Patient’s Diagnostic Journey With Systemic Mastocytosis https://checkrare.com/a-patients-diagnostic-journey-with-systemic-mastocytosis/ Joan Smith, patient with systemic mastocytosis, discusses her diagnostic journey with systemic mastocytosis (SM). Results from the ElevAATe Clinical Trial of Efdoralprin Alfa for Patients With AAT Deficiency https://checkrare.com/results-from-the-elevaate-clinical-trial-of-efdoralprin-alfa-for-patients-with-aat-deficiency/ Alaa Hamed, MD, Global Head of Medical Affairs Rare Diseases at Sanofi, discusses results from the ElevAATe clinical trial of efdoralprin alfa for the treatment of patients with alpha-1 antitrypsin deficiency (AATD). 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. New Treatment Option (SAT-3247) for Duchenne Muscular Dystrophy Shows Promise in Early Phase Trial https://checkrare.com/new-treatment-option-sat-3247-for-duchenne-muscular-dystrophy-shows-promise-in-early-phase-trial/ Wildon Farwell, MD, Chief Medical Officer at Satellos, discusses the safety and efficacy of SAT-3247 to treat adults with Duchenne muscular dystrophy (DMD).