Orphan

FDA approves transthyretin amyloidosis therapy of Akcea, Ionis Patients with an orphan, hereditary disease that is transthyretin amyloidosis with a second new treatment option are available after the Food and Drug Administration approved Akcea Therapeutics and Ionis Pharmaceuticals' Tegsedi.  As Tegsedi is approved,...

Introduction: Any disease that affects less than 200,000 patients or 1 in 1500 is termed as a rare disease. There are around 5000 to 8000 rare disease reported, out which only 10% have cure. According to the European Organization for rare disease, 80% of rare diseases are genetic in nature. Some of the rare disease...