Navigation Links
Drug Discovery in Rare Diseases
Date:9/20/2012

NEW YORK, Sept. 20, 2012 /PRNewswire/ -- Reportlinker.com announces that a new market research report is available in its catalogue:

 

Drug Discovery in Rare Diseases

http://www.reportlinker.com/p0968548/Drug-Discovery-in-Rare-Diseases.html#utm_source=prnewswire&utm_medium=pr&utm_campaign=Drug_Discovery_and_Development

 

In comparison with major diseases, the targeting of rare diseases poses many different challenges, necessitating consideration of bespoke R&D strategies for drug discovery efforts to be successful. This report examines the role that low disease prevalence plays in determining the most suitable R&D path.Understand the growing interest in developing new treatments for rare diseases, and why low patient numbers do not preclude commercial viability.Review the regulatory environment governing the development of orphan drugs in different countries.Identify the key challenges that are presented by low patient prevalences.Assess how the research strategy chosen can be influenced by the disease prevalence.Compare the scenarios in which repurposing of existing drugs offers advantages over the development of novel drugs, and vice versa.Approximately 7,000 rare diseases have been identified, but only a very small proportion of these are currently well treated. Orphans represent a greater proportion of all new BLAs than they do of NMEs submitted as NDAs. Most orphan approvals are not first approvals of new drugs but are new orphan indications for previously approved drugs.It is possible to obtain orphan drug designation for conditions with a total prevalence greater than that defined by legislation, but only if medically justifiable subsets can be defined with a lower (overall) prevalence. Pediatric subsets are most commonly u
'/>"/>

SOURCE Reportlinker
Copyright©2012 PR Newswire.
All rights reserved

Page: 1 2 3 4 5

Related medicine technology :

1. ESTEVE Fosters R & D by Transferring its Center for Drug Discovery to the Science Park of Barcelona (PCB)
2. Initiating Rare Disease Drug Discovery Projects
3. Outsourcing in Drug Discovery: The Contract Research Organization (CRO) Market, 5th Edition
4. AstraZeneca And The Broad Institute Partner To Advance Discovery Of Antibacterial And Antiviral Agents
5. Drug Discovery Technologies, BCC Research
6. Antigen Discovery Inc. Awarded Phase I SBIR Grant to Develop a Fully Integrated Microfluidic Point of Care Diagnostic Device
7. Regulus Therapeutics Forms Strategic Alliance for Discovery, Development and Commercialization of microRNA Therapeutics with AstraZeneca
8. Zenobia Therapeutics Announce A Partnership With The Alzheimers Drug Discovery Foundation
9. Antigen Discovery, Inc. Awarded Phase II SBIR Grant to Identify Biomarkers Associated with Protection against Malaria
10. Omeros Announces Discovery in MASP-2 Program
11. Discovery Labs Receives US Patent for Method to Promote Mucus Clearance with Pulmonary Surfactant
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:1/15/2014)... , Jan. 15, 2014 Reportlinker.com announces ... available in its catalogue: ... Forecast and Market Analysis to 2022 ... Atopic Dermatitis - India Drug Forecast and ...
(Date:1/15/2014)... , Jan. 15, 2014  Zafgen, Inc., a ... needs of severely obese patients, today announced initial ... a selective inhibitor of methionine aminopeptidase 2 (MetAP2), ... form of genetic obesity.  These results showed improvements ...
(Date:1/14/2014)... InformEx, North America,s leading business partnering ... the 30 th edition of its annual conference at ... January 21-24. (Logo: http://photos.prnewswire.com/prnh/20131028/NY05330LOGO-a ) ... and specialty chemical companies will take part in the conference, ...
Breaking Medicine Technology:PharmaPoint: Atopic Dermatitis - India Drug Forecast and Market Analysis to 2022 2PharmaPoint: Atopic Dermatitis - India Drug Forecast and Market Analysis to 2022 3PharmaPoint: Atopic Dermatitis - India Drug Forecast and Market Analysis to 2022 4PharmaPoint: Atopic Dermatitis - India Drug Forecast and Market Analysis to 2022 5Zafgen Announces Initial Results from Phase 2a Study of Beloranib in Patients with Prader-Willi Syndrome 2Zafgen Announces Initial Results from Phase 2a Study of Beloranib in Patients with Prader-Willi Syndrome 3Zafgen Announces Initial Results from Phase 2a Study of Beloranib in Patients with Prader-Willi Syndrome 4Media Advisory: InformEx 2014 Events & Speaker Lineup for 30th Edition Conference in Miami Beach 2
... CVS Caremark Corporation (NYSE: CVS ), today announced ... ended September 30, 2011. (Logo: http://photos.prnewswire.com/prnh/20090226/NE75914LOGO) ... Net revenues increased 12.5% to a record $26.7 billion ... Retail Pharmacy segment revenues increased 3.8%, with same ...
... Bellicum Pharmaceuticals, Inc. today announced publication in ... results using a new therapy that demonstrated rapid and ... Inducible Apoptosis as a Safety Switch for Adoptive Cell ... ™ technology to eliminate donor T cells causing ...
Cached Medicine Technology:CVS Caremark Reports Third Quarter Results 2CVS Caremark Reports Third Quarter Results 3CVS Caremark Reports Third Quarter Results 4CVS Caremark Reports Third Quarter Results 5CVS Caremark Reports Third Quarter Results 6CVS Caremark Reports Third Quarter Results 7CVS Caremark Reports Third Quarter Results 8CVS Caremark Reports Third Quarter Results 9CVS Caremark Reports Third Quarter Results 10CVS Caremark Reports Third Quarter Results 11CVS Caremark Reports Third Quarter Results 12CVS Caremark Reports Third Quarter Results 13CVS Caremark Reports Third Quarter Results 14CVS Caremark Reports Third Quarter Results 15Bellicum Announces Publication in The New England Journal of Medicine Demonstrating Complete Reversal of Serious Immune Condition in Hematopoietic Stem Cell Transplant Patients 2Bellicum Announces Publication in The New England Journal of Medicine Demonstrating Complete Reversal of Serious Immune Condition in Hematopoietic Stem Cell Transplant Patients 3Bellicum Announces Publication in The New England Journal of Medicine Demonstrating Complete Reversal of Serious Immune Condition in Hematopoietic Stem Cell Transplant Patients 4
(Date:4/23/2014)... "simple and accessible detection methods that can rapidly ... of a single cell inside that population has ... Chang Lu. , In the Royal Society of ... announced that he and his coworkers have developed ... of a protein ( Chem. Sci. , 2014, ...
(Date:4/23/2014)... are often displayed in the popular Swedish children,s ... of high-sugar foods. A new study from the ... in children,s TV programmes as well as the ... and weight status. , Steingerdur Olafsdottir,s doctoral thesis ... popular Swedish children,s TV show Bolibompa. Healthy foods ...
(Date:4/23/2014)... led by a researcher from the Cancer Science Institute ... Singapore has identified the cancer specific stem cell which ... of developing new drugs for the treatment of this ... group, led by Dr Chan Shing Leng, Research Assistant ... that a cancer-specific variant of a cell surface protein, ...
(Date:4/23/2014)... in the journal Oncotarget , explored tumour ... or their own DNA signatures within the same ... design effective, targeted treatment strategies. , Firstly they ... and either the KRAS or BRAF mutation. Secondly, ... EGFR gene mutation have that specific mutation present ...
(Date:4/23/2014)... Alzheimer,s disease has been a rapidly evolving pursuit ... latest crook in the research road, scientists have ... proteins associated with the disease. The report, which ... , could have important implications for developing novel ... colleagues explain that for years, research has suggested ...
Breaking Medicine News(10 mins):Health News:Following a protein's travel inside cells is key to improving patient monitoring, drug development 2Health News:High-calorie and low-nutrient foods in kids' TV 2Health News:Scientists identify cancer specific cell for potential treatment of gastric cancer 2Health News:Scientists identify cancer specific cell for potential treatment of gastric cancer 3Health News:Non-uniform genetic mutations identified in lung cancers could lead to targeted treatment 2
... Wash., June 11 Following a two-year clinical ... , Stayhealthy introduced their next generation body composition ... The technology accurately measures body composition, comes in ... a home use individual device, to a commercial ...
... , , Continued Market Share Increase Combined with ... SAN BRUNO, Calif., June 11 Cord Blood Registry ... stem cells from umbilical cord blood, reported solid revenue growth in ... 2008. Earnings for the quarter showed even stronger gains due ...
... ... help parents combat teen drug use, is now available from Bartell Drugs , ... Seattle, CA (Vocus) June 11, 2009 -- HairConfirm, a new ... from Bartell Drugs. The kit detects drug use for up to 90 days by analyzing ...
... ... of British Heart week (8-15th June), Dr Jav Nazemi - CEO of Igennus, ... condition led to the start of his business and the omega-3 EPA formula, ... pence per tub sold this week to the Children,s Heart Federation, in order ...
... ... is a struggle for women of all ages. Despite the turmoil of ... for symptomatic relief of menstrual and menopausal woes. Garden of Life, Inc., ... women of all ages a natural way to balance hormones with the ...
... 10 The following statement was issued Wednesday, June ... Jessie M. Pavlinac, following a press conference by U.S. ... restaurant menu labeling to be included in the Senate,s ... health-care reform legislation. , , "The American Dietetic ...
Cached Medicine News:Health News:New Stayhealthy Body Composition Technology Announced at Microsoft's Connected Health Conference 2Health News:New Stayhealthy Body Composition Technology Announced at Microsoft's Connected Health Conference 3Health News:Cord Blood Registry Maintains Solid Growth in First Quarter 2009 2Health News:Cord Blood Registry Maintains Solid Growth in First Quarter 2009 3Health News:New Home Drug Hair Test Kit Now Available from Bartell Drugs; ,HairConfirm Detects 90-Day Drug Use & Usage Frequency 2Health News:Omega-3 EPA: Treatment for a Heart Condition 2Health News:Omega-3 EPA: Treatment for a Heart Condition 3Health News:Omega-3 EPA: Treatment for a Heart Condition 4Health News:Omega-3 EPA: Treatment for a Heart Condition 5Health News:Omega-3 EPA: Treatment for a Heart Condition 6Health News:MORE WOMEN SHUN HORMONE REPLACEMENT THERAPY Garden of Life Offers a Natural Solution with Oceans 3™ Healthy Hormones™ 2Health News:MORE WOMEN SHUN HORMONE REPLACEMENT THERAPY Garden of Life Offers a Natural Solution with Oceans 3™ Healthy Hormones™ 3Health News:MORE WOMEN SHUN HORMONE REPLACEMENT THERAPY Garden of Life Offers a Natural Solution with Oceans 3™ Healthy Hormones™ 4Health News:Statement by American Dietetic Association Supporting Scientifically Valid, Informative Restaurant Labeling as Part of Health Reform 2
... is a national reference laboratory and a ... development. ARUP offers an extensive test menu ... in clinical and anatomic pathology. Owned by ... include more than half of the nation's ...
... ARUP Laboratories is a national reference laboratory ... research and development. ARUP offers an extensive ... medical tests in clinical and anatomic pathology. ... Laboratories' clients include more than half of ...
... ARUP Laboratories is a national reference laboratory ... research and development. ARUP offers an extensive ... medical tests in clinical and anatomic pathology. ... Laboratories' clients include more than half of ...
... ARUP Laboratories is a national reference laboratory ... research and development. ARUP offers an extensive ... medical tests in clinical and anatomic pathology. ... Laboratories' clients include more than half of ...
Medicine Products: