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:1/22/2015)... For over 20 years, Dr. David Cruz of ... area has treated just about every type of injury that ... And for those same twenty-plus years, the team at Doctors ... with the best possible doctor. With the combined breadth ...
(Date:1/22/2015)... A study recently published in ... Medicine & Science in Sports & Exercise shows ... reduce gastrointestinal (GI) problems associated with physical strain ... the double-blind, placebo-controlled study took 10 grams of ...
(Date:1/22/2015)... 2015 IsoComforter, Inc. has introduced the most ... IsoTube design to treat specific body parts and muscle groups. ... and easy to use patented cold therapy machine with the ... orthopedic and muscle injuries. IsoComforter has become the industry ...
(Date:1/22/2015)... EBSCO Information Services (EBSCO) and the ... an effort to further promote visibility and adoption of the ... made AMA journals available via its subscription services, EBSCO will ... , Long known as both a subscription agency ...
(Date:1/22/2015)... a new blog post presenting a list of vehicles that cannot ... Some types of vehicles cannot be covered under an ordinary auto ... see if their vehicle qualifies for auto insurance coverage. , ... that cannot be covered by a plan. Clients have little options ...
Breaking Medicine News(10 mins):Health News:Industry Leading Chiropractor in Oceanside Joins the Doctors on Liens Network 2Health News:Study Shows Kyowa Hakko’s L-Citrulline May Help Reduce Gastrointestinal Injury During Strenuous Exercise 2Health News:Study Shows Kyowa Hakko’s L-Citrulline May Help Reduce Gastrointestinal Injury During Strenuous Exercise 3Health News:IsoComforter Introduces Best of Class Cold Therapy Product to the Worldwide Market 2Health News:EBSCO Expands Partnership with the American Medical Association 2Health News:EBSCO Expands Partnership with the American Medical Association 3Health News:EBSCO Expands Partnership with the American Medical Association 4Health News:Some Vehicles Cannot Be Covered By An Auto Insurance Policy! 2
... as a normal part of aging. It shouldn,t be. ... a nursing,home, and death. Suicide risk also increases with ... rate in the United States. Depression in the,elderly can ... can be challenging not to mistake them for symptoms ...
... Poster Presented at Annual Meeting of the American Academy of ... ... 11 NeoStrata today released new,clinical research that validates the benefits ... as a complement to,medical anti-aging treatments such as lasers and microdermabrasion. ...
... the UC Davis M.I.N.D. Institute and Center for Childrens ... of mothers of children with autism bind to fetal ... authors also found that the reaction was most common ... autism, which occurs when a period of typical development ...
... Spa is pleased,to announce the appointment of fitness ... "Jeanine brings outstanding credentials to Deerfield,s fitness,program," ... enable her,to fine tune our offerings so guests ... at the spa, but also learn skills to ...
... Only Non-Alcohol Product that can be ... Sprayed Directly into the Face, UNION, ... revolutionary new product for use after a,potential pathogenic exposure, MyClyns(TM), is ... site, added a customer,service center and deployed regional product specialists to ...
... Patent Expands Geographic Coverage, IRVING, ... (OTC Bulletin Board: CARN) today announced ... patent application No. 200600484, titled,"Delivery of ... Anionic,Polysaccharides," relating to the Company,s proprietary ...
Cached Medicine News:Health News:Depression in the Elderly is Complicated but Treatable, From the Harvard Mental Health Letter 2Health News:NeoStrata Research Validates Anti-Aging Benefits of At-Home AHA Peels As Complement to In-Office Non-Invasive Cosmetic Procedures 2Health News:Some cases of autism may be traced to the immune system of mothers during pregnancy 2Health News:Some cases of autism may be traced to the immune system of mothers during pregnancy 3Health News:Some cases of autism may be traced to the immune system of mothers during pregnancy 4Health News:Deerfield Spa Appoints New Fitness Director 2Health News:First Responders Acquire Revolutionary MyClyns(TM) Protective Spray 2Health News:First Responders Acquire Revolutionary MyClyns(TM) Protective Spray 3Health News:Carrington's DelSite Drug Delivery Technology Granted Patent From Eurasian Patent Office 2Health News:Carrington's DelSite Drug Delivery Technology Granted Patent From Eurasian Patent Office 3
(Date:1/23/2015)... 2015  Now available for sale, The Armor1 Ankle Roll ... ankle sprains by cushioning the ankle from an inversion ... the outside of any shoe type and allows the ... offering protection against sprains. With customers in physical therapy, ...
(Date:1/23/2015)... , Jan. 23, 2015  CytoSorbents Corporation (NASDAQ CM: ... CytoSorb® extracorporeal cytokine adsorber to reduce deadly inflammation ... receipt of $385,642, net of transaction costs, in ... Technology Business Tax Certificate Transfer Program sponsored by ...
(Date:1/22/2015)... 2015  ViewRay™, a privately held medical device company, announced ... agreement with ITOCHU Corporation (Tokyo Stock Exchange TYO: 8001) ... treatment of cancer in Japan . ... first and only MRI-guided radiation therapy system that images ...
Breaking Medicine Technology:The Armor1 Ankle Roll Guard is the New Breakthrough Solution for Ankle Sprain Prevention! 2CytoSorbents Receives $385,000 in Non-Dilutive Funding from the New Jersey Technology Business Tax Certificate Transfer Program 2CytoSorbents Receives $385,000 in Non-Dilutive Funding from the New Jersey Technology Business Tax Certificate Transfer Program 3ITOCHU Named the Exclusive Distributor for ViewRay's MRI-Guided Radiation Therapy System in Japan 2
... Defibrillator was designed to be as easy ... and shares many of its features, including ... Philips HeartStart Home Defibrillator is an automated ... home use by untrained users. Although small ...
... imaging and an extraordinary combination of ... choice for the multi-specialty clinical environment. ... SSD-4000s software and hardware allows you ... exams while achieving high-quality results., ,The ...
... is a cost-effective, office-based ultrasound system. It ... quality of our more expensive ProSound models ... maneuvering in small exam rooms., ,The SSD-3500 ... data management system (iDMS) enables easy storage ...
Inquire...
Medicine Products: