Featured Research

from universities, journals, and other organizations

Sanfilippo B: Promising new therapy for devastating genetic disorder

Date:
February 12, 2014
Source:
Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center (LA BioMed)
Summary:
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.

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 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 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.


Story Source:

The above story is based on materials provided by Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center (LA BioMed). Note: Materials may be edited for content and length.


Cite This Page:

Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center (LA BioMed). "Sanfilippo B: Promising new therapy for devastating genetic disorder." ScienceDaily. ScienceDaily, 12 February 2014. <www.sciencedaily.com/releases/2014/02/140212132857.htm>.
Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center (LA BioMed). (2014, February 12). Sanfilippo B: Promising new therapy for devastating genetic disorder. ScienceDaily. Retrieved April 16, 2014 from www.sciencedaily.com/releases/2014/02/140212132857.htm
Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center (LA BioMed). "Sanfilippo B: Promising new therapy for devastating genetic disorder." ScienceDaily. www.sciencedaily.com/releases/2014/02/140212132857.htm (accessed April 16, 2014).

Share This



More Mind & Brain News

Wednesday, April 16, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

Cognitive Function: Is It All Downhill From Age 24?

Cognitive Function: Is It All Downhill From Age 24?

Newsy (Apr. 15, 2014) — A new study out of Canada says cognitive motor performance begins deteriorating around age 24. Video provided by Newsy
Powered by NewsLook.com
App Fights Jet Lag With The Power Of Math

App Fights Jet Lag With The Power Of Math

Newsy (Apr. 13, 2014) — Researchers at the University of Michigan have designed an app to fight jet lag by adjusting your body's light intake. Video provided by Newsy
Powered by NewsLook.com
Treatment Gaps Endangering Cops, Mentally Ill

Treatment Gaps Endangering Cops, Mentally Ill

AP (Apr. 10, 2014) — As states slash funding for mental health services, police officers are interacting more than ever with people suffering from schizophrenia and other serious disorders of the mind. The consequences can be deadly. (April 10) Video provided by AP
Powered by NewsLook.com
Teen Drinking Rates Linked To Alcohol Mentions In Pop Music

Teen Drinking Rates Linked To Alcohol Mentions In Pop Music

Newsy (Apr. 9, 2014) — A University of Pittsburgh study found pop music that mentions alcohol is linked to higher drinking rates among teens. Video provided by Newsy
Powered by NewsLook.com

Search ScienceDaily

Number of stories in archives: 140,361

Find with keyword(s):
 
Enter a keyword or phrase to search ScienceDaily for related topics and research stories.

Save/Print:
Share:  

Breaking News:
from the past week

In Other News

... from NewsDaily.com

Science News

Health News

Environment News

Technology News



Save/Print:
Share:  

Free Subscriptions


Get the latest science news with ScienceDaily's free email newsletters, updated daily and weekly. Or view hourly updated newsfeeds in your RSS reader:

Get Social & Mobile


Keep up to date with the latest news from ScienceDaily via social networks and mobile apps:

Have Feedback?


Tell us what you think of ScienceDaily -- we welcome both positive and negative comments. Have any problems using the site? Questions?
Mobile iPhone Android Web
Follow Facebook Twitter Google+
Subscribe RSS Feeds Email Newsletters
Latest Headlines Health & Medicine Mind & Brain Space & Time Matter & Energy Computers & Math Plants & Animals Earth & Climate Fossils & Ruins