Navigation Links
LA BioMed researchers report on promising new therapy for devastating genetic disorder
Date:2/12/2014

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
lmecoy@labiomed.org
310-546-5860
Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center (LA BioMed)
Source:Eurekalert

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:
*Name:
*Comment:
*Email:
(Date:9/22/2017)... ... September 22, 2017 , ... ... and Richard Pedranti Architect (RPA), a full-service architecture firm focusing on ... Solsken Line of Model Homes . Independently, Ecocor and RPA have extensive ...
(Date:9/22/2017)... Sonora, California (PRWEB) , ... September 22, 2017 ... ... of the inaugural SABRE Safe Is Smart Scholarship Sweepstakes.,  , Molli C., a ... pursuing an MS in Educational Leadership and Administration at St. Thomas University in ...
(Date:9/22/2017)... ... September 22, 2017 , ... “Cursed with a ... obligatory tithe, and the freedom experienced when breaking free from this misconception. “Cursed ... Lysa M. Harrison. , Published by Christian Faith Publishing, Lysa M. Harrison’s new ...
(Date:9/22/2017)... ... September 22, 2017 , ... ... explores the world of Alzheimer’s. Alzheimer’s is a disease estimated to affect the ... to information published by the Alzheimer’s Association, a leading voice in the fight ...
(Date:9/22/2017)... ... ... From Home”: a moving compilation of letters that remind readers of the fact ... the creation of published author, John Allred, a passionate leader of ministry to new ... has traveled and ministered on four continents. , “It is my hope and daily ...
Breaking Medicine News(10 mins):
(Date:9/1/2017)... -- Bayer will present the latest research from across its growing ... 2017 Congress, September 8-12 in Madrid, Spain ... data on Bayer,s marketed portfolio and late-stage compounds as well ... "We value the opportunity to meet ... ESMO," said Carsten Brunn , Head of Pharmaceuticals, Americas ...
(Date:8/29/2017)... HAMPTON, Va. , Aug. 29, 2017 ivWatch, LLC, ... of peripheral IV infiltrations, announced it has been awarded an Innovative ... improvement company in the country. ... A continuous monitoring device to aid in the early detection of ... The ...
(Date:8/28/2017)... CHICAGO , Aug. 28, 2017 Hill-Rom Holdings, ... Healthcare Conference on Monday, September 11, 2017, in ... and chief executive officer, is scheduled to present at 11:05 ... can be accessed at http://ir.hill-rom.com/events.cfm . A recorded replay ... live event through December 10, 2017. ...
Breaking Medicine Technology: