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:7/24/2017)... (PRWEB) , ... July 24, 2017 , ... ... $1,000 total in scholarship awards to be awarded annually to and divided between ... an interest in bringing awareness to Amazonian plant medicine. To apply for the ...
(Date:7/24/2017)... ... July 24, 2017 , ... Independence Blue ... hand-in-hand on an Innovation Collaboration program, have signed a five-year agreement that will ... of the care members and patients receive. The agreement also signifies Jefferson Health ...
(Date:7/24/2017)... Pass, OR (PRWEB) , ... July 24, 2017 , ... ... Global Climate Change and Your Health on VoiceAmerica sponsored by Nature’s Tears® EyeMist® recently ... about the rise of myopia in China. , The article by Zidor Aldama described ...
(Date:7/24/2017)... ... July 24, 2017 , ... ... that they have enrolled over 100,000 children in their treatment program. Clubfoot is ... year in areas where treatment is limited or non-existent. Without intervention, these children ...
(Date:7/24/2017)... Long Island (PRWEB) , ... July 24, 2017 ... ... of a new office at 2307 Bellmore Avenue, Bellmore, New York, (516) 784-5858. ... dermatologist Jordan Fabrikant, M.D. and physician assistants Lori Friedman and Fruma Leah Wiederman. ...
Breaking Medicine News(10 mins):
(Date:7/13/2017)... RK Logistics Group, Inc. was awarded California ... Fremont, CA headquarters facility where it provides full-spectrum ... San Jose for hi-tech, pharmaceutical and biomedical devices ... its Fremont Innovation District, is excited to strategically partner with ... the hundreds of biotech, pharma and biomed companies throughout the ...
(Date:7/11/2017)... Research Corp., also known as ,The Epigenetics Company, announced today the release ... manner using the myDNAge ™ test. Based on Horvath,s Clock, so ... and biostatistics at the David Geffen School of Medicine at UCLA ... DNAge ™ technology is used to analyze DNA methylation patterns of ... ...
(Date:7/11/2017)... Fla. , July 11, 2017  Dr. Echenberg, founder of Echenberg ... therapy program to patients who suffer from painful intercourse and other painful ... and vaginal atrophy due to menopause. ... VuVatech LLC ... , created by Sarasota, Florida -based start-up company, VuVatech ...
Breaking Medicine Technology: