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Latest Mucopolysaccharidosis Stories

2010-12-29 07:46:00

(Ivanhoe Newswire) "“ Researchers believe that newborn screening and early treatment for a rare genetic disorder, mucopolysaccharidosis type I (MPS I), is needed so that enzyme replacement therapy can begin at birth and eliminate all the symptoms associated with MPS I. MPS I is an inherited genetic disorder that affects many body systems. It is caused by a defect in the gene that makes an enzyme called alpha-L-iduronidase. Because of this defect, cells either produce the enzyme in low...

2010-12-14 08:00:00

NOVATO, Calif., Dec. 14, 2010 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the Medicines and Healthcare Products Regulatory Agency (MHRA) in the UK has completed review of the Clinical Trial Application (CTA) and issued a notice of acceptance for the Phase 3 trial of N-acetylgalactosamine 6-sulfatase (GALNS) for the treatment of Mucopolysaccharidosis IVA (MPS IVA). The company expects to initiate the Phase 3 pivotal trial early in the first...

2010-12-07 08:00:00

NOVATO, Calif., Dec. 7, 2010 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that Joshua Grass, Vice President of Corporate and Business Development of BioMarin, will present a company update at the Deutsche Bank 2010 BioFEST Conference in Boston on Tuesday, December 14, 2010 at 9:00 a.m. ET. Interested parties may access a live audio webcast of the conference call via the investor section of the BioMarin website, www.BMRN.com. A replay of the call will be...

2010-12-02 23:38:38

A new study offers hope for children born with a rare genetic disease, according to a paper published by the American Association for the Advancement of Science. The research was led by Dr. Matthew Ellinwood, a veterinarian and animal science professor at Iowa State University, in collaboration with Dr. Patricia Dickson at the Harbor-UCLA Medical Center, with colleagues at the Iowa State College of Veterinary Medicine, the University of Tennessee, St. Louis University and the University of...

2010-12-02 20:50:55

Scientists from The University of Manchester say a naturally occurring chemical found in soy could prove to be an effective new treatment for a fatal genetic disease that affects children. Dr Brian Bigger, from the University's MPS Stem Cell Research Laboratory, found that genistein "“ derived from soya beans and licensed in the US as an osteoporosis drug "“ had a dramatic effect on mice suffering from the human childhood disease Sanfilippo. "Sanfilippo is an untreatable...

2010-12-02 15:00:00

NOVATO, Calif., Dec. 2, 2010 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the Medicines and Healthcare Products Regulatory Agency (MHRA) in the UK has completed review of the Clinical Trial Application (CTA) and has issued a notice of acceptance for BMN 673 for genetically-defined cancers. The company expects to initiate a Phase 1/2 trial by the first quarter of 2011. "BMN 673 has been proven to be highly active in mouse xenograft models of human cancer...

2010-12-02 00:52:40

In a study that supports the need for newborn screening and early treatment for a rare genetic disorder, researchers at Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center (LA BioMed) and Iowa State University found enzyme replacement therapy beginning at birth eliminated almost all of the symptoms associated with mucopolysaccharidosis type I, or MPS I, in animal models. The study, which was published today in the journal, Science Translational Medicine, is the first to...

2010-11-29 08:00:00

NOVATO, Calif., Nov. 29, 2010 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that Jeffrey Cooper, Chief Financial Officer of BioMarin, will present a company update at the Piper Jaffray Healthcare Conference in New York City on Tuesday, November 30, 2010 at 1:00 p.m. ET. Interested parties may access a live audio webcast of the conference call via the investor section of the BioMarin website, www.BMRN.com. A replay of the call will be archived on the site...

2010-11-09 08:00:00

NOVATO, Calif., Nov. 9, 2010 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that six holders of its 2.50% Convertible Senior Subordinated Notes due 2013 have agreed to exchange $87.0 million in aggregate principal amount of the notes for approximately 5.25 million shares of the Company's common stock. The notes represented approximately 50% of the previously outstanding principal amount. "This partial exchange allows us to reduce the amount of debt...

2010-10-19 07:00:00

NOVATO, Calif., Oct. 19 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced its program for BMN-111, a peptide therapeutic for the treatment of achondroplasia. BioMarin plans to file an IND in the fourth quarter of 2011 and to initiate a Phase 1 clinical trial by the first quarter of 2012. BMN-111, for the treatment of achondroplasia, is an analog of C-type Natriuretic Peptide (CNP), a small cyclic peptide that is a positive regulator of bone growth. It...


Word of the Day
mundungus
  • A stinking tobacco.
  • Offal; waste animal product; organic matter unfit for consumption.
This word comes from the Spanish 'mondongo,' tripe, entrails.