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Last updated on April 20, 2014 at 0:02 EDT

Latest Lysosomal storage disease Stories

2014-02-04 12:28:02

CINCINNATI, Feb. 4, 2014 /PRNewswire-USNewswire/ -- Researchers used blood platelets and bone marrow cells to deliver potentially curative gene therapy to mouse models of the human genetic disorder Hurler syndrome - an often fatal condition that causes organ damage and other medical complications. (Logo: http://photos.prnewswire.com/prnh/20110406/MM79025LOGO) Scientists from Cincinnati Children's Hospital Medical Center and the National Institute of Neurological Disorders and...

2013-12-03 23:02:00

St. Jude Children’s Research Hospital scientists identify enzyme that could lead to better diagnosis and treatment of Alzheimer’s disease and related disorders. Memphis, Tenn. (PRWEB) December 03, 2013 St. Jude Children’s Research Hospital scientists have identified an enzyme that can halt or possibly even reverse the build-up of toxic protein fragments known as plaques in the brains of mice with Alzheimer’s disease. The research appeared in a recent edition of the scientific...

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....

2013-05-01 16:31:58

U.S. commercial launch anticipated by end of Q2 2013 NOVATO, Calif., May 1, 2013 /PRNewswire/ -- Raptor Pharmaceutical Corp. ("Raptor" or the "Company") (Nasdaq: RPTP), today announced that the U.S. Food and Drug Administration (FDA) has approved PROCYSBI(TM) (cysteamine bitartrate) delayed release capsules for the treatment of nephropathic cystinosis in adults and children 6 years and older. In a Phase 3 study, PROCYSBI showed consistent cystine depletion over the full 12-hour...

2013-04-30 16:29:08

U.S. commercial launch anticipated by end of Q2 2013 NOVATO, Calif., April 30, 2013 /PRNewswire/ -- Raptor Pharmaceutical Corp. ("Raptor" or the "Company") (Nasdaq: RPTP), today announced that the U.S. Food and Drug Administration (FDA) has approved PROCYSBI(TM) (cysteamine bitartrate) delayed release capsules for the treatment of nephropathic cystinosis in adults and children 6 years and older. In a Phase 3 study, PROCYSBI showed consistent cystine depletion over the full...

2013-02-19 08:32:40

LEXINGTON, Massachusetts, February 19, 2013 /PRNewswire/ -- Shire plc (LSE: SHP, NASDAQ: SHPG), announces that the Lysosomal Disease Network (LDN) presented Shire Human Genetic Therapies (HGT) with the first ever Novel Treatment Award for VPRIV on February 14th, at its 9th Annual WORLD Symposium. The LDN have launched this new award, which will be presented annually, to recognize new drugs or therapies that have made a considerable contribution in the area of lysosomal...

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...

2013-01-28 08:28:37

WASHINGTON, Jan. 28, 2013 /PRNewswire/ -- Today ten families of patients with a rare disease called cystinosis and three foundations supporting cystinosis research tell their stories to policymakers on Capitol Hill in an effort to educate lawmakers about rare diseases like cystinosis and how upcoming federal spending decisions could impact the availability of medicines for rare diseases. Cystinosis is a metabolic lysosomal storage disease that is diagnosed in infancy and often results in...

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...

2012-02-29 04:00:00

Rare Pictures of Hope(TM) Campaign Launched to Spotlight Those Suffering from Rare Diseases NIH and FDA Unite To Hold Rare Disease Awareness and Educational Events DANA POINT, Calif., Feb. 29, 2012 /PRNewswire-USNewswire/ -- Hope - It's In Our Genes.(TM) Today, on the rarest of calendar days, thousands of organizations and millions of rare disease advocates are observing the fifth annual World Rare Disease Day. The R.A.R.E. Project, a leading rare disease patient advocacy...