Over the last 11 years, Rare Disease Day has become iconic as the global campaign for raising awareness of rare diseases among politicians, clinicians, researchers, and pharmaceutical companies. This year’s theme is a testament to the challenges patients and families...
Representatives from the ECDGA attended the Endocrinology Society’s annual conference in New Orleans to raise awareness of ECD among attending endocrinology specialists.
The US Food and Drug Administration has granted a Breakthrough Therapy Designation to cobimetinib in the treatment of patients with histiocytic neoplasms (Erdheim-Chester Disease, Rosai-Dorfman, Langerhans Histiocytosis), who do not bear the BRAF V600 mutation. This...