Recent News Stories
MPS Society in the UK Commits $250,000 toward MPS III ResearchThe MPS Society in UK recently committed $250,000 to fund the research of Professor Grzegorz Wegrzyn in Gdansk, Poland, "Development of Gene Expression-Targeted Isoflavone Therapy (GET IT) for Mucopolysaccharidosis Type III". Below is the statement from the UK MPS Society about this research. More...
TKT's I2S for Hunter Syndrome Recieves Office of Orphan Products Grant
Transkaryotic Therapies, Inc. (Nasdaq: TKTX) today announced that it has received a Development Grant from the FDA's Office of Orphan Products Development (OOPD) for iduronate-2-sulfatase (I2S), TKT's investigational enzyme replacement therapy for the treatment of Hunter syndrome, also known as MPS II, a rare, fatal disease. More...
Scientific protocol for treating children with severe MPS I with a combination of Stem Cell Transplantation and Enzyme Replacement Therapy at the University of MinnesotaThe University of Minnesota has been a pioneer in the treatment of children with severe MPS I, also called Hurler syndrome, and other storage disorders using stem cell transplantation (i.e., bone marrow or cord blood transplantation). To date, approximately 100 transplants have been performed at the University of Minnesota for Hurler syndrome alone. More...
New MPS I RegistryAccess to information is critical to providing the best care to patients with MPS I however information on the disease is limited because of its rarity. Now there is a resource which your physician or health care professional can use which is dedicated to improving the understanding of MPS I disease. More... |