The 2016 Rare Diseases & Orphan Products Breakthrough Summit, a two-day conference led by the National Organization for Rare Disorders (NORD), was held right outside of Washington D.C. in October...
Eteplirsen (to be marketed as ExonDys 51), an antisense oligonucleotide developed by Sarepta Therapeutics, was just recently approved by the FDA as the first therapy targeting the underlying cause of...
/ 2 years ago
Xiidra is only the second dry eye disease prescription drug to be approved in the United States. Remarkably, FDA’s approval of Shire’s Xiidra (lifitegrast ophthalmic solution) comes more than a...
With the approaching launch of two novel therapies that have shown substantial clinical promise to date, the coming decade is set to be a very exciting time for individuals suffering from chronic and/...
Orkambi is the first therapy addressing the underlying cause of the disease in cystic fibrosis (CF) patients with two copies of the F508del mutation. Orkambi launched in the United States in July 2015...
What is Duchenne Muscular Dystrophy? Duchenne muscular dystrophy (DMD), the most common form of childhood muscular dystrophy, results from the absence or reduction in levels of the dystrophin protein...
/ 3 years ago