Current Status Not Enrolled Price Free Get Started Login to Enroll Course Resources Information Gaucher Disease: Epidemiology and Pathophysiology Program: Challenges of Diagnosing and Caring for an Individual with Gaucher Disease Slides Gaucher Disease Module 1 slides Resources National Library of Medicine: Gaucher Disease https://ghr.nlm.nih.gov/condition/gaucher-disease National Institute of…
Leslie Edwin, President of the CSRF, and Gretchen Jordan, Associate Director of the CSRF, discuss their personal diagnostic journeys.
Mario Maldonado, MD, Head of Clinical Development for Global Endocrinology at Recordati Rare Diseases, discusses current treatment options for patients with Cushing’s syndrome.
Hana Weltin, Intern at Beyond the Diagnosis, discusses how she advocates for herself and others in the rare disease space.
Frank Rivera, Founder of Sarcoidosis of Long Island, discusses how his organization fulfills the unmet needs of sarcoidosis patients.
Richard Wasserman, MD, PhD, discusses the FDA expanded label approval for Xembify for primary immunodeficiencies (PIDs).
Prem Subramanian, MD, PhD, discusses long-term data testing teprotumumab in patients with thyroid eye disease (TED).
Ben Zimmer, CEO of Priovant Therapeutics, discusses the clinical trial testing investigational therapy for dermatomyositis, brepocitinib.
Hypoparathyroidism is a rare endocrine disorder characterized by a deficiency of parathyroid hormone (PTH).
Pisit “Duke” Pitukcheewanont, MD, Lumos Pharma, discusses a phase 2 trial evaluating LUM-201 for treatment of growth hormone deficiency.
Hideki Garren, MD, PhD, discusses the pathophysiology of AL amyloidosis and the mechanism of action of investigational drug birtamimab.
