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It also adds a biologic medicine in an orphan disease with strong intellectual property protection through 2027 and further diversifies net sales.
The federal government also has avenues through which funds are available; the FDA issues orphan grants, and the Rare Disease Clinical Research Network at NIH funds 22 orphan disease consortia, each focusing on a single rare disorder or family of like rare disorders.
The initial chapters of the report provide an orientation of orphan disease dealmaking and its associated business and partnering activities.
It covers legislation and the definition of orphan disease in different countries, the main steps in drug development, the characteristics of rare diseases, the role of patient network and advocacy groups, organizations and networks dedicated to rare diseases and orphan drugs, policies and research funding in the US and European Union, designing clinical trials, and market access procedures.
A RARE disease, which may also be referred to as an orphan disease, is one which affects a small percentage of the population.
Tokyo, Feb 15, 2013 - (JCN) - Daiichi Sankyo will collaborate with a new company, Orphan Disease Treatment Institute, in the development of a treatment for Duchenne muscular dystrophy (DMD) with the active ingredient ENA oligonucleotide, a modified nucleic acid made using proprietary technology owned by Daiichi Sankyo.
That view is echoed by Yann Le Cam, head of the EU-wide orphan disease patients' group Eurordis, who wants to see more flexible licensing that would allow a new drug to come to market earlier with limited distribution while more data is collected.
Lipoprotein lipase deficiency is a rare disease, a so-called orphan disease, that affects one or two of every million people.
Establishing a dedicated operation in the United States strengthens our presence in the largest healthcare market in the world and Massachusetts is the capital of orphan disease drug development," said Philip Astley-Sparke Non-Executive Chairman of the Board of Oxyrane.
This partnership brings Amgen, a world leader in human genetic target validation, together with Boston Children's Hospital's Division of Pain Medicine, the first and most active pediatric pain programme in the world, and its Manton Center for Orphan Disease Research.
18 June 2013 - Several pharmaceutical firms have shown interest in buying US biotechnology company ViroPharma Inc (NASDAQ:VPHM), although the rare and orphan disease treatment maker is not looking for a buyer, three insiders told Reuters.
The NCOD also asked for more federal funds for orphan disease research, speedier FDA approval of medications for victims of rare diseases, and relocation of the orphan drug program from FDA to a central office in HHS.