
The FDA approved tiratricol (Emcitate) for peripheral thyrotoxicosis in MCT8 deficiency, the first approved therapy for the rare disorder.
New international pediatric HAE guideline recommends oral sebetralstat as first-line acute therapy for adolescents 12 years and older.
A newly published international guideline on the diagnosis and management of pediatric hereditary angioedema (HAE) provides updated, evidence-based recommendations for children and adolescents with HAE due to C1 inhibitor deficiency (HAE-C1INH), including a strong recommendation for sebetralstat as a first-line option for acute treatment in adolescents 12 years and older.1,2






