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™.

Related Articles


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 November 28, 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 November 28, 2014).

Share This


More From ScienceDaily



More Mind & Brain News

Friday, November 28, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

Tryptophan Isn't Making You Sleepy On Thanksgiving

Tryptophan Isn't Making You Sleepy On Thanksgiving

Newsy (Nov. 27, 2014) — Tryptophan, a chemical found naturally in turkey meat, gets blamed for sleepiness after Thanksgiving meals. But science points to other culprits. Video provided by Newsy
Powered by NewsLook.com
Are Female Bosses More Likely To Be Depressed?

Are Female Bosses More Likely To Be Depressed?

Newsy (Nov. 24, 2014) — A new study links greater authority with increased depressive symptoms among women in the workplace. Video provided by Newsy
Powered by NewsLook.com
Winter Can Cause Depression — Here's How To Combat It

Winter Can Cause Depression — Here's How To Combat It

Newsy (Nov. 23, 2014) — Millions of American suffer from seasonal depression every year. It can lead to adverse health effects, but there are ways to ease symptoms. Video provided by Newsy
Powered by NewsLook.com
Could Your Genes Be The Reason You're Single?

Could Your Genes Be The Reason You're Single?

Newsy (Nov. 21, 2014) — Researchers in Beijing discovered a gene called 5-HTA1, and carriers are reportedly 20 percent more likely to be single. 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:

Strange & Offbeat Stories

 

Health & Medicine

Mind & Brain

Living & Well

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