Rare Disease Report

Endocrinology

The Phase 2 CARDINAL study is evaluating bardoxolone methyl (bardoxolone) in patients with chronic kidney disease because of Alport syndrome.
André the Giant, examines the life and work history of the WWE Hall of Famer. Unbeknownst to many, his size was the result of gigantism, a disease caused by excess growth hormone. The condition led to his eventual diagnosis with acromegaly, a rare endocrine disorder.
When taken early in a pregnancy, mifepristone can end a pregnancy that is less than 49 days along. Another notable trait of the drug, though, is its ability to control the high blood sugar in patients with Cushing syndrome who have type 2 diabetes and have either failed surgery or are not candidates for it.
TED, also known as Graves’ eye disease, is an autoimmune disorder best characterized by the appearance of buldging eyes.
The survey consisted of 12 multiple-choice questions to determine the physical, emotional, and informational challenges these patients face.
Lutathera, the 177-Lu somatostatin analog, is a new form of targeted therapy.
In February 2016, the FDA approved Afinitor (everolimus) for treating neuroendocrine tumors (NETs) of gastrointestinal or lung origin.
Diarrhea is a common problem for patients with short bowel syndrome.
A Prescription Drug User Fee Act (PDUFA) date is set for December 30, 2017.
Allena announced that the FDA has granted Orphan Drug Designation to its compound, ALLN-177, for the treatment of primary hyperoxaluria.
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