Navigation Links
LA BioMed researchers report on promising new therapy for devastating genetic disorder

LOS ANGELES (Feb. 12, 2014) A promising new therapy has for the first time reduced damage to the brain that can be caused by Sanfilippo B (MPS IIIB), a rare and devastating genetic disease, Los Angeles Biomedical Research Institute (LA BioMed) researchers will report today in a presentation at the Lysosomal Disease Network's 10th Annual WORLD Symposium.

Sanfilippo B syndrome is a lysosomal storage disease belonging to the group of mucopolysaccharidoses (MPS) that is characterized by severe and rapid intellectual deterioration. LA BioMed pioneered the research that led to the first enzyme replacement therapy for MPS I, Aldurazyme, which has saved hundreds of lives in the nearly 11 years since it was approved by the Food and Drug Administration.

LA BioMed's MPS Research Laboratory reported the results of its latest study for treating Sanfilippo B syndrome in a disease model that employed recombinant human "NAGLU-IGF2" (a novel fusion protein of alpha-N-acetyglucosaminidase, or NAGLU, coupled to a peptide derived from the insulin-like growth factor, IGF2). The fusion protein was produced by BioMarin Pharmaceutical Inc.

LA BioMed researcher Shih-hsin Kan reported that the laboratory's tests found NAGLU-IGF2 is taken up by neurons in the disease model, which then reduces brain damage by reversing heparan sulfate storage one of the causes of damage to the brain in patients with Sanfilippo B syndrome. The researchers concluded that NAGLU-IGF2 can be the basis for enzyme replacement therapy for Sanfilippo B syndrome.

"This is an exciting new development that needs to be examined further to determine its effectiveness in patients with Sanfilippo B syndrome who currently have no therapies available to reduce the damage to their brains," said Patricia Dickson, MD, director of the MPS Research Laboratory at LA BioMed. "We look forward to continuing our partnership with BioMarin in developing therapies for MPS."

BioMarin announced Tuesday that it has developed BMN 250, a manufactured form of NAGLU-IGF2, for the treatment of Sanfilippo B syndrome and expects to initiate clinical studies with BMN 250 in mid-2015.


Contact: Laura Mecoy
Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center (LA BioMed)

Related medicine news :

1. Pitts Center for Medical Innovation announces pilot funding for biomedical technology
2. New biomedical diagnostics using personalized 3-D imaging
3. NCCS and Clearbridge Biomedics open the regions first circulating tumor cell center
4. LA BioMed study finds daily antibiotics most effective in preventing recurrent urinary tract infection
5. A New Player In Biomedical Equipment Opens Headquarters in San Diego
6. Four Researchers from Icahn School of Medicine at Mount Sinai Among the 400 Most Highly Influential Biomedical Researchers in the World
7. CHI and PwC US Release 2014 California Biomedical Industry Report
8. New materials with potential biomedical applications
9. California Healthcare Institute to Release 2014 California Biomedical Industry Report
10. Animal, human health benefits anticipated from universitys premier biomedical instrument
11. MU receives $1.1 million training grant to increase diversity in biomedical sciences
Post Your Comments:
(Date:11/30/2015)... ... November 30, 2015 , ... ... four states in the U.S. require dental technicians to be certified or obtain ... industry, NADL created the “What’s In Your Mouth?” campaign to inform dentists that ...
(Date:11/30/2015)... MN (PRWEB) , ... November 30, 2015 , ... ... at . The directory is specialized and only includes chiropractic clinics ... to find a competent and trustworthy alternative health practitioner when back pain sets ...
(Date:11/30/2015)... ... November 30, 2015 , ... ... experience this summer, ushering in a new era of publicly accessible automated technology. ... shuttle, will continue to offer guests an up-close look at the shuttle at ...
(Date:11/30/2015)... (PRWEB) , ... November 30, 2015 , ... ... and fun atmosphere for Halloween festivities, the Word of Life Christian Church of ... board game, and featuring a giant 1.25 ton pile of candy dubbed “Candy ...
(Date:11/30/2015)... (PRWEB) , ... November 30, 2015 , ... ... community across the United States to access life-saving information provided directly ... Mesothelioma will consist of three individual conferences in three major cities: Houston, ...
Breaking Medicine News(10 mins):
(Date:11/30/2015)... 30, 2015 Nevro Corp. (NYSE: NVRO ), ... solutions for the treatment of chronic pain, today announced that ... Patent and Trademark Office (USPTO) has denied instituting an ... (the ,102 patent).  --> ... Scientific Corporation filed two petitions challenging the validity of certain ...
(Date:11/30/2015)... SAN FRANCISCO , Nov. 30, 2015 ... and Designers of Things (DoT ) co-located events ... Printing and the Internet of Things, will draw more ... San Jose Convention Center. The events, combined ... their latest technologies. --> ...
(Date:11/30/2015)... and BOCA RATON, Fla. ... for the Commercialization of Public Research (the Institute) ... with KynderMed , a medical device start-up company ... supports new company creation based on publicly-funded research, and ... Florida -based universities and research institutions. ...
Breaking Medicine Technology: