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Cystinosis in Medical News

Raptor Pharmaceuticals Corp. to Present at 2009 Cystinosis Research Network Family Conference

... development of the product candidate, at the cystinosis Research Network ("CRN") Family Conference, taking place July 16-18, 2009 in Atlanta, GA. cystinosis is a rare genetic disease characterized by the ... in the first two years of life. Failure to treat cystinosis can cause serious health consequences, including ...

2009 Science Report on Genetic Disease Released by Cystinosis Research Foundation

... Researchers of the genetic disease cystinosis report encouraging news. The cystinosis Research Foundation has published the third ... Report, which is designed to provide updates on cystinosis research under way and includes new abstracts of ...

Cystinosis Research Network Unveils New Communication Strategy and Website

... cystinosis Research Network, a non-profit organization ... Lake Forest, IL (PRWEB) January 29, 2009 -- The cystinosis Research Network, a non-profit organization aimed ... the organization's efficacy as the voice of the cystinosis community. President and Executive Director ...

Raptor Pharmaceuticals Corp. and TorreyPines Therapeutics, Inc. Announce Merger Agreement

... populations where it can have the greatest potential impact. Raptor currently has product candidates in clinical development to treat nephropathic cystinosis , non-alcoholic steatohepatitis ("NASH") , Huntington's Disease ("HD") , and aldehyde dehydrogenase ("ALDH2") deficiency . Raptor's ...
Cystinosis in Medical Technology

Raptor Pharmaceuticals to Collaborate with Centre Hospitalier Universitaire d'Angers for Phase II Clinical Trial in Huntington's Disease

... Medicines Agency ("EMEA") to treat nephropathic cystinosis ("cystinosis"), a rare, genetic lysosomal storage ... to initiate a pivotal study of DR Cysteamine in cystinosis patients in 2009. About Raptor Pharmaceuticals ... ("ALDH2") deficiency, nephropathic cystinosis and non-alcoholic steatohepatitis ("NASH"). ...

Raptor Pharmaceuticals Appoints Dr. Patrice P. Rioux as Chief Medical Officer

... Cysteamine ("DR Cysteamine") for the potential treatment of nephropathic cystinosis ("cystinosis"), non-alcoholic steatohepatitis ("NASH") and Huntington's ... Company expects to conduct its pivotal clinical study for DR Cysteamine in cystinosis in the second half of 2009 and file an NDA in 2010. In February, ...

Raptor Pharmaceuticals Announces Completion of Patient Enrollment in Phase 2a Clinical Trial in Non-Alcoholic Steatohepatitis (NASH)

... ("FDA") and European Medicines Agency ("EMEA") to treat nephropathic cystinosis ("cystinosis"), has demonstrated potential efficacy in preclinical and ... is to leverage cysteamine's known safety profile for the treatment of cystinosis and potential efficacy in several other indications. In this case, ...

Raptor Pharmaceuticals Announces Phase 2a Clinical Trial Initiation in Non-Alcoholic Steatohepatitis (NASH)

... ("FDA") and European Medicines Agency ("EMEA") to treat nephropathic cystinosis ("cystinosis"), a rare and genetic lysosomal storage disease. Raptor is ... Raptor's ability to develop DR Cysteamine for the treatment of NASH, cystinosis and other indications; Raptor's ability to formulate and manufacture DR ...

Raptor Pharmaceuticals Corp. Announces Positive Phase IIa Clinical Study Results for Convivia(TM) Program

... the greatest potential impact. Raptor is currently running clinical trials in patients with aldehyde dehydrogenase ("ALDH2") deficiency, nephropathic cystinosis and non-alcoholic steatohepatitis ("NASH"). Raptor's preclinical programs are based upon bioengineered novel drug candidates and drug-targeting ...

Raptor Pharmaceuticals Enters Collaboration Agreement with UC San Diego in Liver Disease

... involved in clinical trials in patients with aldehyde dehydrogenase ("ALDH2") deficiency as part of Raptor's Convivia(TM) program and nephropathic cystinosis as part of Raptor's DR Cysteamine program. Raptor's preclinical division bioengineers novel drug candidates and drug-targeting platforms derived from ...
Cystinosis in Medical Definition

Pathology

... , Propionic acidemia , Methylmalonic acidemia , Isovaleric acidemia , 3-Methylcrotonyl-CoA carboxylase deficiency ) - Transport ( Cystinuria , cystinosis , Hartnup disease ) - Sulfur ( Homocystinuria , Cystathioninuria ) - Urea cycle ( Argininosuccinic aciduria , Citrullinemia , ...
Cystinosis in Medical Dictionary

Cystinosis

... cystinosis follows a pattern of autosomal recessive ... they also have a normal gene to compensate. cystinosis - an inherited disorder whereby the kidney s ... which binds excess copper in the blood . cystinosis is treated with cysteamine, which removes ...

Fructose

... RTA can also result from inherited disorders that disrupt the body's normal breakdown and use of nutrients. Examples include the rare disease cystinosis (in which cystine crystals are deposited in bones and other tissue s), hereditary fructose ... Read the ingredient list for packaged foods ...

Metabolism

... less than 1% of the T3 is free. Cystine is made by cells during metabolism . Normally the amino acid is carried out of the cell but in cystinosis the cystine cannot be transported out. Thyroid cancer : Cancer of the gland in front of the neck that normally produces thyroid hormone ...
Cystinosis in Biological News

Brain-nourishing molecule may predict schizophrenia relapse

... Exercise can increase BDNF levels. Two drugs currently on the market for other medical conditions erythropoietin for anemia and cysteamine for cystinosis can as well. Dr. Pillai hopes his laboratory studies of these drugs will soon result in an adjunct therapy that can help patients avoid devastating ...
Cystinosis in Biological Technology

Raptor Pharmaceuticals Doses First Patient in Phase 2b Clinical Trial of DR Cysteamine for Cystinosis

... ("DR Cysteamine") capsules in nephropathic cystinosis ("cystinosis"), a rare genetic lysosomal storage ... The Phase 2b clinical trial will enroll up to six cystinosis patients with a history of good compliance using ... study with a pivotal, Phase 3 clinical study in cystinosis patients anticipated to commence in the fourth ...

Raptor Pharmaceuticals Corp. Announces Presentation at 5th International Cystinosis Conference 2008

... cystinosis Foundation Ireland to Host Event June 27-28, ... Raptor's clinical division, will present at the cystinosis Foundation Ireland's 5th International cystinosis Conference to be held June 27-28, 2008 at ...

Raptor Pharmaceuticals Acquires Orphan Clinical Program

... in a clinical trial generously supported by the cystinosis Research Foundation of Irvine, CA. The active ... the lead clinical investigator for the ongoing cystinosis trial. "Although cysteamine bitartrate has been ... "Through the generous and untiring support of the cystinosis Research Foundation over the past five years, we ...

Vista Partners Updates Coverage on Raptor Pharmaceuticals Corp. (OTC Bulletin Board: RPTP)

... FDA approval of Delayed Release (DR) Cysteamine for the treatment of cystinosis in 2010," stated Ross Silver, Director of Research for Vista Partners. ... deficiency as part of Raptor's Convivia(TM) program and nephropathic cystinosis as part of Raptor's DR Cysteamine program. Raptor's preclinical division ...

Raptor Pharmaceuticals Corp. Enters Agreements to Advance Clinical Pipeline

... is currently approved by the FDA for the management of nephropathic cystinosis ("cystinosis"), a genetic lysosomal storage disease characterized by the ... rights to Cysteamine in NASH complement our license to DR Cysteamine for cystinosis and neurodegenerative diseases. In addition to our ongoing clinical trial ...

Raptor Pharmaceuticals Closes $10 Million Private Placement

... approval and commercialization. Raptor's initial clinical programs include the treatment of aldehyde dehydrogenase ("ALDH2") deficiency, nephropathic cystinosis and non-alcoholic steatohepatitis ("NASH"). Raptor preclinical programs target cancer, neurodegenerative disorders and infectious diseases. ...

Raptor Pharmaceuticals Corp. To Present at BIO 2008 International Convention

... currently approved indications. Bennu's initial clinical programs include the treatment of aldehyde dehydrogenase ("ALDH2") deficiency, nephropathic cystinosis and NASH. For additional information, please visit http://www.raptorpharma.com . FORWARD LOOKING STATEMENTS This document contains ...

Raptor Pharmaceuticals Corp. Receives FDA Orphan Drug Designation for Cysteamine in Huntington's Disease

... currently approved indications. Bennu's initial clinical programs include the treatment of aldehyde dehydrogenase ("ALDH2") deficiency, nephropathic cystinosis and NASH. For additional information, please visit http://www.raptorpharma.com . FORWARD LOOKING STATEMENTS This ...
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