Navigation Links
NIH clinical trial begins for treatment of rare, fatal neurological disorder
Date:1/23/2013

A clinical trial to evaluate a drug candidate called cyclodextrin as a possible treatment for Niemann-Pick disease type C1 (NPC), a rare and fatal genetic disease, will start today, researchers announced. Scientists from the NIH's National Center for Advancing Translational Sciences (NCATS) and the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) will conduct the clinical trial at the NIH Clinical Center. Reaching this trial stage required collaboration among government, industry, patient advocacy groups and academic researchers.

No therapies approved by the U.S Food and Drug Administration are available to treat NPC. The disease is characterized by the inability of cells to metabolize and dispose of cholesterol and lipids. It causes excessive amounts of cholesterol to accumulate within the liver, spleen and brain. NPC leads to progressive impairment of motor and intellectual function in early childhood. In childhood onset cases, life expectancy does not normally exceed a patient's teenage years.

"A crucial part of the NCATS mission is to collaborate within and beyond the NIH on projects to improve and accelerate the translational research process and deliver tangible improvements in human health," said NCATS Director Christopher P. Austin, M.D. "The cyclodextrin project is an important step in the development of both a potential treatment for a devastating disease that ravages the bodies and minds of its victims and a more efficient way to do translational projects."

In 2009, the NIH Therapeutics for Rare and Neglected Diseases (TRND) program, which is now led by NCATS, selected NPC cyclodextrin as one of its initial pilot projects to repurpose cyclodextrin from its conventional use as an ingredient in other drugs to a therapeutic for this rare disorder. TRND researchers work with project collaborators to conduct preclinical studies advancing potential treatments for rare and neglected diseases
'/>"/>

Contact: NCATS Office of Communications
info@ncats.nih.gov
301-435-0888
NIH/National Center for Advancing Translational Sciences (NCATS)
Source:Eurekalert

Page: 1 2 3

Related medicine news :

1. ASH international clinical collaboration replicates high cure rate of APL in developing countries
2. Life Support Technologies Group Appoints Edward Golembe, MD as Co-Clinical Director
3. Life Support Technologies Group Appoints Scott Gorenstein, MD as Co-Clinical Director
4. Declining access to electroconvulsive therapy: A clinical choice or an economic one?
5. Regenerative medicine: Clinical trials launched for the treatment of delayed union fractures
6. Cancer Genome Institute at Fox Chase among first to offer clinical blueprint of cancer genes
7. When will genomic research translate into clinical care -- and at what cost?
8. Clinical Nutrition, 2nd Edition
9. BioPharm Systems to Host Webinar on Using iHelp in Oracle’s Siebel Clinical Trial Management System
10. Sozo Nutritionals Announces Release of Svetol® Based Natural Weight Loss Product Backed By Several Clinical Studies Showing Remarkable Weight Loss Benefits
11. APS announces inaugural issue of new journal, Clinical Psychological Science
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:5/24/2015)... New York (PRWEB) May 24, 2015 ... treatment lawsuits ( http://www.testosteronelawsuithub.com/ ) has scheduled its ... progress of the multidistrict litigation. According to court ... District of Illinois, the Conference will be convened ... re: Testosterone Replacement Therapy Product Liability Litigation - ...
(Date:5/24/2015)... 2015 Indiana Fiber Network, LLC (IFN), ... Telecom of America, Inc. (MTA) has selected IFN as ... Childs, IFN Enterprise Sales Manager, fiber route diversity and ... fiber transport provides MTA with the diverse fiber transport ... 1 Internet and voice carriers located at Henry Street ...
(Date:5/24/2015)... Lake Geneva, WI (PRWEB) May 24, ... Bea Mercy Health System CEO ... their principal and honored at the ... event held April 15 at Geneva ... include (by school): ,     -Badger High ...
(Date:5/23/2015)... FL (PRWEB) May 23, 2015 On Saturday, ... build a new playground for Branches Florida City exactly five ... former play space in 2010. , A large fire tore ... playground on May 23, 2010 in the middle of the ... The fire was later declared arson even though it is ...
(Date:5/23/2015)... Dr. David Benvenuti, an experienced plastic surgeon who practices ... article to his website that takes an in-depth look ... in the fascinating blog, he has recently had several young ... as “gauging,” the technique involves placing larger and larger gauge ... eventually stretch the holes out to incredible sizes. As the ...
Breaking Medicine News(10 mins):Health News:Testosterone Lawsuit Attorneys at Bernstein Liebhard LLP Look Ahead to June Conference in Federal Multidistrict Litigation 2Health News:Testosterone Lawsuit Attorneys at Bernstein Liebhard LLP Look Ahead to June Conference in Federal Multidistrict Litigation 3Health News:Indiana Fiber Network, LLC Enables Midwest Telecom of America with Fiber Route Diversity 2Health News:Mercy Foundation Honors 16 Students for Making a Difference - Mercy Health System Javon Bea 2Health News:Five Years After Fire, Branches Rebuilds Playground 2Health News:Dr. Benvenuti, a Plastic Surgeon in Newport Beach, CA, Posts New Article about Repairing Ear Gauges 2
... are uncommon as children enter adolescence, parasomnias present in ... a research abstract that will be presented Monday, June ... the 24th annual meeting of the Associated Professional Sleep ... persistence after five years was 29 percent for children ...
... Newly reported results from a major clinical trial ... chemotherapy for women with advanced ovarian cancer and ... afterwards significantly extends progression-free survival. Women receiving the ... disease for 14.1 months, compared to 10.3 months ...
... found compound slowed drug-resistant medulloblastoma in children , ... has found that a targeted treatment for medulloblastoma -- ... may one day be able to treat drug-resistant forms ... patients currently survive medulloblastoma," said Dr. Amar Gajjar, lead ...
... survival, researchers report , SATURDAY, June 5 (HealthDay News) -- ... first in its class, improved survival by 68 percent in ... parts of the body. , This is big news in ... to budge, despite numerous efforts to come up with an ...
... CHICAGO - Selenium, a supplement taken daily by millions in ... diseases, has proven to be of no benefit in reducing ... recurrence or second primary malignancy, according to results of an ... study, initiated by the Eastern Cooperative Oncology Group, were presented ...
... ... Day 2010 GreatDad Recommends Award and Mr. Dad Seal of Approval Recipients , ... San Francisco, Calif. (PRWEB) June 5, 2010 ... for a real treat. Presented by Mr. Dad and GreatDad.com, the results of the Fathers ...
Cached Medicine News:Health News:Childhood parasomnias such as sleepwalking and bedwetting may persist into adolescence 2Health News:New treatment regimen shown effective against advanced ovarian cancer 2Health News:Targeted Therapy Shows Promise Against Deadly Brain Cancer 2Health News:Novel Drug Combats Advanced Melanoma 2Health News:Novel Drug Combats Advanced Melanoma 3Health News:Selenium shows no benefit in prevention of lung cancer 2Health News:Selenium shows no benefit in prevention of lung cancer 3Health News:Mr. Dad and GreatDad.com Salute Fathers and Families with This Spring's ‘Seal of Approval' Picks 2Health News:Mr. Dad and GreatDad.com Salute Fathers and Families with This Spring's ‘Seal of Approval' Picks 3Health News:Mr. Dad and GreatDad.com Salute Fathers and Families with This Spring's ‘Seal of Approval' Picks 4
(Date:5/22/2015)... , May 22, 2015 The leading ... $13 billion global cardiac rhythm management device market ... found that Medtronic, St. Jude Medical, Boston Scientific ... of this market segment. The healthcare market research ... , uses 2015 as a base year, providing ...
(Date:5/22/2015)... , May 22, 2015  RegeneRx Biopharmaceuticals, Inc. ... tissue protection, repair and regeneration, today announced that ... scheduled to present a corporate overview at two upcoming ... at the SeeThruEquity 4 th Annual Microcap Conference ... York City at 11:00 am EDT.  A ...
(Date:5/22/2015)... OF PRUSSIA, Pa. , May 22, 2015  The ... Behring its 2015 Corporate Leadership Award as recognition for ... improving the care of the bleeding disorders community.  The award ... Director, CSL Limited, during the NHF Annual Spring Soiree in ... "The National Hemophilia Foundation is dedicated to finding ...
Breaking Medicine Technology:Kalorama: Top Companies Dominate Cardiac Defibrillator Market 2Kalorama: Top Companies Dominate Cardiac Defibrillator Market 3Kalorama: Top Companies Dominate Cardiac Defibrillator Market 4RegeneRx to Present at 2 Investor Conferences 2RegeneRx to Present at 2 Investor Conferences 3National Hemophilia Foundation Honors CSL Behring with Corporate Leadership Award 2National Hemophilia Foundation Honors CSL Behring with Corporate Leadership Award 3National Hemophilia Foundation Honors CSL Behring with Corporate Leadership Award 4
... YORK, May 13, 2011 Duane Reade Holdings, Inc. today ... new fundraising record for the 26th annual AIDS Walk New ... raises funds for Gay Men,s Health Crisis (GMHC) and 41 ... care, and more to people living with HIV/AIDS, as well ...
... For the second year in a row Royce Colors - ... a supplier recognition from Carestream Health, the world,s leading manufacturer ... which supplies a complete line of Medical and Dental Imaging ... 2010 Supplier of the Year , Carestream,s top supplier ...
Cached Medicine Technology:Duane Reade Celebrates Fourth Year Supporting AIDS Walk New York With Record Donation 2Duane Reade Celebrates Fourth Year Supporting AIDS Walk New York With Record Donation 3Royce Receives Coveted Medical Products Award 2
The AP250 Auto Projector is a remote-controlled, programmable, random-access, high-resolution, ophthalmic projector....
The Onar®Lp -QP test system is an in vitro test for the quantitative diagnosis of legionella in clinical samples....
The GoodKnight® 425 Bi-Level® device is suitable for travel and includes features such as automatic altitude compensation, optional cigarette lighter adapter and portable battery pack...
... The BiPAP Pro with Bi-Flex bi-level system, ... that the BiPAP Pro takes to change ... Digital Auto-Trak Sensitivity™ which automatically triggers the ... pressures based on the patients breathing rhythm. ...
Medicine Products: