One of the main problems is, because of the small number of people with a particular orphan disease
, running meaningful clinical trials is very difficult.
Daiichi Sankyo and Orphan Disease
Treatment Institute will jointly conduct clinical and non-clinical studies with the cooperation and support of these two professors with the aim of achieving POC.
Charlie is an expert in LSDs with over 10 years' drug development experience in the rare and orphan disease
The 1985 amendments to the Act also established a National Commission on Orphan Diseases
(NCOD) to evaluate government research activities on rare diseases.
PharmGPS(TM) Orphan Disease
Suite BioXcel's proprietary Orphan Disease
Suite encompasses more than 9,000 rare & ultra-rare diseases, 4,000 -5,000 associated genes, 1,500 disease pathways, and distinct target-indication tiles for antibody, protein, RNA, small molecule and gene therapy modalities.
Daiichi Sankyo Company, Limited (hereafter, Daiichi Sankyo) announced today the opening of the office of the Orphan Disease
Treatment Institute Co.
GBI Research, the leading business intelligence provider, has released its latest research, Orphan Disease
Therapeutics in Genetic Disorders to 2018 - Emerging Agents in Cystic Fibrosis Offer Strong Opportunities for Investment and Licensing Activity, which provides insights into the global orphan diseases
in genetic disorders therapeutics market, including market forecasts until 2018.
BRANFORD, Connecticut, June 11, 2014 /PRNewswire/ -- BioXcel Corporation, announced today its first-in-class cloud based Pharma big-data analytics platform, PharmGPS(TM) now has a dedicated Orphan Disease
suite to aid in the discovery, development, licensing and commercialization of drugs for the approximately 7,000 Rare and Ultra Rare Diseases.
The new report* shows that the relatively unsatisfied market for orphan disease
drugs could prove to be a big hit for pharmaceutical companies able to improve upon the limited options currently available to patients.
A popular section with readers; chapter 3 covers the average financial deal terms for deals signed in the orphan disease
field with a stage of development announced.
uniQure has developed Glybera as a therapy for patients with the genetic disorder lipoprotein lipase deficiency, an orphan disease
GBI Research, the leading business intelligence provider, has released its latest research Orphan Diseases
Therapeutics in Oncology to 2018 - Strongly Diversified Developmental Pipelines Indicate Long-Term Growth Potential despite Moderate Scope of Current Late Stage Molecules, which provides insights into the Oncology orphan disease
therapeutics market until 2018.