Rare Diseases
Commentary
Growth hormone therapy for certain children may help them reach their potential
When should parents consider growth hormone therapy? In what cases is it warranted for children? Dr. Jessica Sparks Lilley discusses the need for...
Latest News
Lipedema: A potentially devastating, often unrecognized disease
“If patients with lipedema gain weight, the problem becomes even worse,” Dr. C. William Hanke said.
News
FDA approves levoketoconazole for Cushing syndrome
The drug, which inhibits cortisol synthesis, is seen as addressing an unmet need.
Latest News
FDA approves first once-weekly growth hormone for children
The heiGHt trial demonstrated noninferiority of lonapegsomatropin to somatropin daily injections.
Conference Coverage
Two treatments show early promise for hypothalamic obesity
Conference Coverage
Hyperphagia, anxiety eased with carbetocin in patients with Prader-Willi syndrome
Conference Coverage
Success in achondroplasia spurs testing vosoritide in more growth disorders
The quality of growth achieved with vosoritide in achondroplasia is leading to studies in more growth disorders.
News
FDA clears device to remove dead pancreatic tissue
News
FDA clears first drug for rare genetic causes of severe obesity
Setmelanotide (Imcivree) is indicated for weight management in adults and children as young as 6 years with severe obesity driven by genetic...
Conference Coverage
Burosumab is a ‘game changer,’ effective in all subgroups of XLH
The recently approved treatment improved serum phosphorus and key measures of pain and stiffness in subgroups of adults with this rare form of...
Feature
CDER chief reflects on advances in rare diseases
“People always call me fearless, but I feel like I just state the facts,” Dr. Janet Woodcock said.