OPKO Health announced their phase 3, double-blind, placebo-controlled study investigating long-acting human growth hormone product (hGH-CTP) in adults with growth hormone deficiency (GHD) did not meet its primary endpoint (change in trunk fat mass).
Video highlights from the Rare Diseases Heroes Event in Arlington, VA.
Stay At Home Chef Rachel Farnsworth took some time away for posting cooking tips to address the fact that she has Addison’s disease and people should not judge her by her looks
In this interview during the Rare Disease Heroes Award Ceremony, Paul Thornton, MD, of Cook Children's in Fort Worth TX explains the pathophysiology of congenital hyperinsulinism as well as how it is currently treated. Dr Thornton also discusses some future therapies that are begin developed for this rare condition.
In this interview during the Rare Disease Heroes Award Ceremony, Lee and Zachary Parker talk about Zachary's diagnosis of hyperinsulinism
The Rare Disease Heroes Award recognizes and celebrates those hospitals that are advancing the management of rare diseases.
POEMS syndrome (aka osteosclerotic myeloma, Takatsuki syndrome, and Crow-Fukase syndrome) is a rare paraneoplastic syndrome. A new study shows treatment can improve sexual function in POEM patients.
Five year data showing the efficacy and safety of Strensiq (asfotase alfa) to treat children with hypophosphatasia (HPP) was published this week in the Journal of Clinical Investigations.
When patients are close to death, it is important to weigh the benefits and potential drawbacks of continued treatment. Dr Yuhua Bao of Weill Cornell Medical College and colleagues have highlighted the importance of this approach for stage IV pancreatic patients.
More interim data on the use of Afinitor (everolimus) for advanced neuroendocrine tumors (NETs) was presented at ASCO.