Hunter syndrome

Jul 08, 2025

Merck's WINREVAIR Gets FDA Priority Review for Pulmonary Arterial Hypertension Merck, also known as MSD outside the U.S. and Canada, announced that the FDA has accepted and granted Priority Review to the supplemental Biologics License Application (sBLA) for WINREVAIR (sotatercept-csrk). The application seeks to ...

May 21, 2025

Lysosomal Storage Disorders, or LSDs, are rare genetic conditions that may not make headlines, but they affect thousands worldwide, often with life-changing consequences. LSDs happen when the body’s lysosomes, tiny cellular recycling centers, fail to break down waste materials due to missing or faulty enzymes. This...

Mar 19, 2025

Mucopolysaccharidosis (MPS) is a group of rare genetic disorders affecting roughly 1 in 25,000 births, yet its impact is profound. Caused by enzyme deficiencies, MPS leads to the buildup of complex sugars in cells, resulting in progressive damage to the heart, bones, brain, and other organs. Symptoms can vary widel...

May 22, 2020

The Hunter Syndrome Market is expected to grow with CAGR of 7.5% in the coming years in the 7MM. Occurring in approximately 1 in 100,000 to 1 in 170,000 male births, Hunter Syndrome, is an X-linked disorder, of carbohydrate metabolism that occurs almost exclusively in males, with females as the carriers of the d...

Oct 13, 2015

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...