Navigation Links
Raptor Pharmaceuticals Corp. Receives FDA Orphan Drug Designation for Cysteamine in Huntington's Disease
Date:6/9/2008

Potential Efficacy in Huntington's Disease Already Demonstrated

NOVATO, Calif., June 9 /PRNewswire-FirstCall/ -- Raptor Pharmaceuticals Corp. ("Raptor" or the "Company") (OTC Bulletin Board: RPTP), today announced that the U.S. Food and Drug Administration ("FDA") has granted orphan drug designation for cysteamine bitartrate ("cysteamine") for the treatment of Huntington's disease ("HD"). Cysteamine is currently approved by the FDA and European Medicines Agency ("EMEA") to treat nephropathic cystinosis ("cystinosis"), a rare lysosomal storage disease. Preclinical results suggest that cysteamine has neuroprotective effects that could potentially help treat HD. Raptor's clinical development subsidiary, Bennu Pharmaceuticals Inc. ("Bennu"), plans to evaluate cysteamine in patients with HD.

HD is a rare and hereditary neurological disease thought to occur in approximately 1 out of every 20,000 people resulting in around 20,000 patients in the U.S. presently. There is currently no drug available that targets the unique molecular defect that is believed to cause HD, a progressive disease that often leads to death within 15 to 20 years after diagnosis. HD is caused by a defective gene resulting in the degeneration of certain nerve cells in the brain. The disease is characterized by uncontrollable movements and mood swings or depression, followed by dementia. Preclinical findings on cysteamine's mechanism of action suggest that it has neuroprotective effects by increasing the levels of BDNF, a neuroprotective neurotrophic factor, in the brain of HD mice. BDNF levels may also be a potential biomarker of efficacy for cysteamine in HD, an important development issue when a therapy is used to prevent or slow clinical manifestation of a disorder.

The 1983 Orphan Drug Act provides important economic incentives to encourage companies to develop potential therapies for the diagnosis, prevention and/or treatment of rare, serious diseases affecting 200,0
'/>"/>

SOURCE Raptor Pharmaceuticals Corp.
Copyright©2008 PR Newswire.
All rights reserved

Page: 1 2 3 4 5

Related biology technology :

1. Raptor Pharmaceuticals Acquires Orphan Clinical Program
2. Raptor Pharmaceuticals Corp. Enters Agreements to Advance Clinical Pipeline
3. Vion Pharmaceuticals Pays Interest on 7.75% Convertible Senior Notes
4. China Biopharmaceuticals Holdings Announces Second Quarter 2007 Financial Results
5. Vion Pharmaceuticals Completes Accrual of 85 Patients to its Pivotal Phase II Trial of Cloretazine(R) (VNP40101M) in Elderly AML
6. XTL Biopharmaceuticals Announces Financial Results for the Six Months Ended June 30, 2007
7. Cadence Pharmaceuticals Reports Second Quarter 2007 Financial Results
8. Nabi Biopharmaceuticals to Present at Upcoming Industry Conferences
9. Anadys Pharmaceuticals Appoints Steve Worland, Ph.D. as President and Chief Executive Officer
10. Poniard Pharmaceuticals Announces Participation in Upcoming Conferences
11. NovaQuest Advances Virtual Development Model With Strategic Investment in TOPIGEN Pharmaceuticals
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:9/22/2014)... Sept. 22, 2014  Neogen Corporation (Nasdaq: ... first quarter of its 2015 fiscal year, which ended ... to net income of $7,839,000 in the first quarter ... effective Oct. 30, 2013, earnings per share in the ... ago. First quarter revenues increased 15% to ...
(Date:9/22/2014)... -- The US markets on Friday, September ... Dow Jones Industrial Average finished at 17,279.74, up 0.08% ... The S&P 500 finished the session 0.05% lower at ... sectors finished on a positive note. The S&P 500 ... up 0.10%, with the index gaining 6.95% in the ...
(Date:9/22/2014)... , Sept. 22, 2014  Spherix Incorporated (SPEX) -- ... monetization of intellectual property, today announced that the United States ... the Company in the month of September that is part ... The issued patent is: USRE45,121 , Application number: ... , Filing date: Dec 27, 2012 The ...
(Date:9/21/2014)... September 21, 2014 Chemical Sciences ... challenges such as clean air and safe water, ... natural products. Chemists, environmentalists, food scientists, educators, and ... research in the field of Chemistry for the ... Sciences is one of the fastest emerging Sciences, ...
Breaking Biology Technology:Neogen reports 13% net income increase 2Neogen reports 13% net income increase 3Neogen reports 13% net income increase 4Neogen reports 13% net income increase 5Neogen reports 13% net income increase 6Neogen reports 13% net income increase 7Stock Updates on Biotech Industry - Array BioPharma, Enzon Pharma, Alkermes, Amarin, and Synergy Pharma 2Stock Updates on Biotech Industry - Array BioPharma, Enzon Pharma, Alkermes, Amarin, and Synergy Pharma 3Stock Updates on Biotech Industry - Array BioPharma, Enzon Pharma, Alkermes, Amarin, and Synergy Pharma 4Stock Updates on Biotech Industry - Array BioPharma, Enzon Pharma, Alkermes, Amarin, and Synergy Pharma 5Spherix Notified by United States Patent & Trademark Office of New Standard Essential Patent Issued to Spherix 2Spherix Notified by United States Patent & Trademark Office of New Standard Essential Patent Issued to Spherix 3Chemistry for Global Development during the Open Access Week 2Chemistry for Global Development during the Open Access Week 3
... New Report Added in ResearchMoz Reports ... Spending Estimates and Forecasts) The ... past ten years suggests the amount of vaccine production ... adapt to match a revived industry. This ...
... 15, 2012 Healthcare organizations are becoming more efficient ... creating a more connected patient care environment. But to ... information, especially the electronic medical record (EMR) and enterprise ... Software, a leader in ECM for healthcare , ...
... improve infrared photodetector technology that is critical to national ... in commercial applications and consumer products. A significant ... journal Applied Physics Letters . It ... more effectively by using certain materials arranged in specific ...
Cached Biology Technology:ResearchMoz: Vaccine Production Market (Trends, Techniques, Key Players, Spending Estimates and Forecasts) 2ResearchMoz: Vaccine Production Market (Trends, Techniques, Key Players, Spending Estimates and Forecasts) 3ResearchMoz: Vaccine Production Market (Trends, Techniques, Key Players, Spending Estimates and Forecasts) 4ResearchMoz: Vaccine Production Market (Trends, Techniques, Key Players, Spending Estimates and Forecasts) 5Johns Hopkins Health System is 100th healthcare Provider to Integrate OnBase With Leading Electronic Medical Record System 2Johns Hopkins Health System is 100th healthcare Provider to Integrate OnBase With Leading Electronic Medical Record System 3New nano-material combinations produce leap in infrared technology 2New nano-material combinations produce leap in infrared technology 3
(Date:9/19/2014)... not evolve for the sake of fairness per se ... cooperation, so say Frans de Waal, PhD, and Sarah ... aversion (IA), which is defined as a negative reaction ... Science . , Their conclusion comes after the ... their hypothesis that it is the evolution of forestalling ...
(Date:9/19/2014)... study shows that legume plants regulate their symbiotic ... that are transmitted through the plant structure from ... of bacteria-holding nodules in the roots. This collaborative ... Institute for Basic Biology, the Graduate University for ... Sustainable Resource Science in Japan. , Legumes, ...
(Date:9/18/2014)... A sleep-promoting circuit located deep in the primitive brainstem ... by researchers at Harvard School of Medicine and the ... this is only the second "sleep node" identified in ... necessary and sufficient to produce deep sleep. , Published ... study demonstrates that fully half of all of the ...
Breaking Biology News(10 mins):Evolution of responses to (un)fairness 2Evolution of responses to (un)fairness 3For legume plants, a new route from shoot to root 2No sedative necessary: Scientists discover new 'sleep node' in the brain 2
... sunlight decreases the toxicity of golden algae, which kills millions ... While golden algae is primarily a coastal species, it has ... and Lake Waco in Central Texas, and Lake Granbury in ... production, but new research from Baylor scientists shows that sunlight ...
... International and American Associations for Dental Research (IADR/AADR) have ... and Pulp Regeneration," held in Geneva, Switzerland, July 2010. ... and Regeneration Group, and they are published in volume ... an E-supplement to the Journal of Dental Research ...
... CAMBRIDGE, Mass. -- The first organisms to emerge ... skills, arming themselves with tough exteriors to weather a ... Smith College have discovered hundreds of microscopic fossils in ... time when the planet emerged from a global glaciation, ...
Cached Biology News:IADR/AADR publish proceedings from Symposium on Tissue Injury and Pulp Regeneration 2MIT research: Life after 'Snowball Earth' 2MIT research: Life after 'Snowball Earth' 3
Panomer 9 oligodeoxynucleotides are random primers that are labeled at the 5' end with our proprietary QSY 7 dye and they are useful for assessing microarray spot morphology, hybridization potential ...
... l-[U-14C]methionine, 1.85 MBq, 50 uCi. Aqueous ... > 1.85 GBq/mg atom carbon, > ... of Ala, Arg, Asp, Glu, Gly, ... Ser, Thr, Tyr and Val.1.85 MBq/ml, ...
Topoisomerase I, isolated from wheat germ, is an enzyme capable of removing negative supercoils from covalently closed circular DNA (1)....
Request Info...
Biology Products: