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:4/29/2016)... ... April 29, 2016 , ... In its efforts of bringing quality senior care ... franchise development and expansion as it welcomes Ferdie Vasquez of the new 1Heart Caregiver ... his 32 years of working in various industries. He had been a Franchise ...
(Date:4/28/2016)... ... 2016 , ... Sublime Beauty® is offering a special deal on its webstore. , Buy 1 ... is applied to the product with lesser value. In addition, a free bonus of ... more in products. , "So many women (men, too) love our products and get good ...
(Date:4/28/2016)... (PRWEB) , ... April 28, 2016 , ... ProText Layouts ... Final Cut Pro X . With ProText Layouts, video editors can create ... to match the length and scale of texts. Creating text-based videos have never ...
(Date:4/28/2016)... ... , ... Head Over Heels Athletic Arts’ gymnast, Brooklyn Varize, has qualified to ... at the University of Montana on April 28-May 1. Varize will compete on Saturday ... order to qualify, Varize needed to place top seven all-around in her respective age ...
(Date:4/28/2016)... ... 2016 , ... The ARRS 2016 Annual Meeting ... from around the globe who attended a week’s worth of cutting-edge scientific presentations, ... included the introduction of the 2016 ARRS Distinguished Educator, Dr. Gerald F. Abbott, ...
Breaking Medicine News(10 mins):
(Date:4/28/2016)... 2016 Treato , the ... announced today that it has been named a Cool ... in Life Sciences, 2016, Stephen Davies , ... focuses on life-science- oriented analytics, algorithms and smart machine ... doctors, confirm medication ingestion, and analyze unstructured information.   ...
(Date:4/28/2016)... 28, 2016 Oramed Pharmaceuticals Inc. (NASDAQ: ... company focused on the development of oral drug delivery systems, ... PIONEERS 2016 conference, presented by Joseph Gunnar & ... New York . Nadav Kidron , CEO ... Presentation Details:   PIONEERS 2016, ...
(Date:4/28/2016)... TapImmune,Inc. (TPIV), a clinical-stage immune-oncology company ... and vaccines for the treatment of cancer & metastatic disease, ... Growth Capital Expo to be held on May 3 ... in Las Vegas, Nevada.  The Company presentation will be ... by Dr. John N. Bonfiglio a TapImmune Board ...
Breaking Medicine Technology: