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Last updated on April 17, 2014 at 16:26 EDT

Latest Mucopolysaccharidosis Stories

2014-03-31 09:52:21

New insight has been gained into treating an inherited disorder that creates serious neurological and behavioral disabilities in children and usually leads to death in the teen years. In a recent study into the effects of human umbilical cord blood mononuclear cells (hUCB MNCs) when they are injected to counter the symptoms and progression of Sanfilippo syndrome type III B (MPS III B), researchers found that repeated injections into laboratory mice modeled with the disorder had clear...

2014-03-11 04:21:27

BARCELONA, Spain, March 11, 2014 /PRNewswire/ -- - This gene therapy program is being developed in a public-private partnership between ESTEVE and the research team of Dr. Fatima Bosch at the Center for Biotechnology and Gene Therapy of the Universitat Autonoma de Barcelona (UAB) - ESTEVE announce the signing of agreements with REGENX and GENETHON which will allow it to initiate its phase I/II clinical trial in 2015 -...

2013-07-18 12:28:16

SANTA MONICA, Calif., July 18, 2013 /PRNewswire/ -- ARMAGEN announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to its lead product AGT-182 for the treatment of mucopolysaccharidosis type II (also known as Hunter syndrome or MPS II.) Hunter syndrome is a rare, genetic lysosomal storage disease caused by a deficient or absent enzyme, iduronate-2-sulfatase. It is a life-threatening disease affecting children as young as 2 years of age....

2013-02-04 16:25:54

CINCINNATI, Feb. 4, 2013 /PRNewswire-USNewswire/ -- Researchers have overcome a major challenge to treating brain diseases by engineering an experimental molecular therapy that crosses the blood-brain barrier to reverse neurological lysosomal storage disease in mice. (Logo: http://photos.prnewswire.com/prnh/20110406/MM79025LOGO) Posted online in PNAS Early Edition on Feb. 4, the study was led by scientists at Cincinnati Children's Hospital Medical Center. "This study provides a...

BioMarin Studies Treatment Of MPS IVA
2012-11-08 07:05:03

Connie K. Ho for redOrbit.com — Your Universe Online Advances in medical technology have made it possible for new treatments to be developed. One such example of the development of treatments is work done by BioMarin Pharmaceutical Inc., a company focused on developing and commercializing biopharmaceuticals for serious diseases and other medical conditions. Having met the primary endpoint of change in a six minute distance walk as compared to a placebo at 24 weeks for participants...

2012-03-28 02:25:38

NOVATO, Calif., March 28, 2012 /PRNewswire/ -- Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced that the European Medicines Agency (EMA) has granted orphan drug designations for sialic acid for the treatment of hereditary inclusion body myopathy (HIBM) and recombinant human beta-glucuronidase for the treatment of mucopolysaccharidosis type 7 (MPS 7). Orphan designation in the European Union...

2012-02-28 05:00:00

NOVATO, Calif., Feb. 28, 2012 /PRNewswire/ -- Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced that the FDA Office of Orphan Products Development has granted orphan drug designation for UX003 for the treatment of MPS 7. MPS 7 is an extremely rare autosomal recessive lysosomal storage disorder characterized by a deficiency of the lysosomal enzyme Beta-glucuronidase, required for the degradation...

2012-01-25 08:00:00

DANA POINT, Calif., Jan. 25, 2012 /PRNewswire-USNewswire/ -- Rare Disease affects 350 million people worldwide, consisting of 7,000+ identified diseases and disorders. Currently less than 5% of all rare diseases have any type of therapy or treatment, and much of the early stage research is frequently seed-funded by parent advocates, advocacy organizations and non-profit groups. There is an important new trend in rare disease research where a group of rare disease advocates...

2012-01-09 11:29:34

A team of scientists, led by researchers at the University of California, San Diego School of Medicine and Zacharon Pharmaceuticals, have developed a simple, reliable test for identifying biomarkers for mucopolysaccharidoses (MPS), a group of inherited metabolic disorders that are currently diagnosed in patients only after symptoms have become serious and the damage possibly irreversible. The findings will be published online January 8 in the journal Nature Chemical Biology. MPS is...

2012-01-05 07:45:00

NOVATO, Calif., Jan. 5, 2012 /PRNewswire/ -- Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced it has in-licensed an enzyme replacement therapy program from St. Louis University to treat mucopolysaccharidosis type 7 (MPS 7). The in-licensed program is a treatment for an ultra-rare genetic, metabolic disorder that results from the deficiency of the beta-glucuronidase...