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:10/13/2017)... ... October 13, 2017 , ... Yisrayl Hawkins, Pastor and Overseer at The ... the most popular and least understood books in the Holy Scriptures, Revelation. The Book ... have baffled scholars for centuries. Many have tossed it off as mere rubbish, but ...
(Date:10/13/2017)... ... October 13, 2017 , ... “America On The Brink”: ... “America On The Brink” is the creation of published author, William Nowers. ... As a WWII veteran, he spent thirty years in the Navy. Following ...
(Date:10/12/2017)... ... October 12, 2017 , ... The company has developed ... consumer and regulatory authorities worldwide. From Children’s to Adults 50+, every formula has ... highest standard. , These products are also: Gluten Free, Non-GMO, Vegan, Soy ...
(Date:10/12/2017)... Rockville, Maryland (PRWEB) , ... October 12, 2017 ... ... a magnetic drug delivery system that we intend to develop to enable prevention ... regimens can lead to severe hearing loss, especially in pediatric patients. For cisplatin, ...
(Date:10/12/2017)... FL (PRWEB) , ... October 12, 2017 , ... On ... holding a treadmill relay – Miles by Moonlight to raise money for the American ... $300 or more. , Teams will work together to keep their treadmills moving ...
Breaking Medicine News(10 mins):
(Date:9/27/2017)... their devotion to personalized service, SMP Pharmacy Solutions announces their ... South Florida Business Journal,s 50 Fastest-Growing Companies, and listed for ... specialty pharmacy has found its niche.  To that end, the ... by SFBJ as the 2017 Power Leader in Health Care. ... award in October, Bardisa said of the three achievements, "It,s ...
(Date:9/25/2017)... 25, 2017  EpiVax, Inc., a leader in ... immune-engineering today announced the launch of EpiVax Oncology ... personalized therapeutic cancer vaccines. EpiVax has provided $500,000 ... to enabling technologies to the new precision immunotherapy ... EpiVax Oncology as Chief Executive Officer. Gad brings ...
(Date:9/22/2017)... Sept. 22, 2017 AVACEN Medical (AVACEN) announced ... now successfully helping those with the widespread pain associated ... diagnosed Amanda in Essex, England ... my hair, experiencing no sleep at all, tremendous pain, ... I cannot recommend [the AVACEN 100] enough, how this ...
Breaking Medicine Technology: