• She is a member of the National Organization for Rare Disorders and the Drug Information Association. (xtalks.com)
  • For the same decade the compound annual growth rate (CAGR) of the orphan drugs was an "impressive 25.8 percent, compared to only 20.1 percent for a matched control group of non-orphan drugs. (wikipedia.org)
  • Founded in Trondheim in 2007 the company has been chartered by the Norwegian Ministry of Health and Care Services to improve global access to orphan medicines. (wikipedia.org)
  • Orphan drugs are medicines that are commercially unavailable because of high research costs and a low potential for profit. (europa.eu)
  • In recent years, patient advocacy groups have expanded their influence over the drug development continuum from discovery to approval to market entry. (xtalks.com)
  • Whilst expansion into MDS is integral to realizing the full commercial potential of omacetaxine, we remain focused on our primary objective of seeking regulatory approval for the drug in CML patients with the T315I mutation. (medicalnewstoday.com)
  • Moreover, we suggest that orphan drugs have greater profitability when considered in the full context of developmental drivers including government financial incentives, smaller clinical trial sizes, shorter clinical trial times and higher rates of regulatory success. (wikipedia.org)
  • The global orphan drugs market report offers in-depth analysis of the market size (revenue), market share, major market segments, different geographic regions, forecast for the next five years, key market players, and premium industry trends. (whatech.com)
  • This growth is attributed to the changing government policies and the rising demand for the orphan drugs from these regions. (whatech.com)
  • Peppermint could provide two harvests per growing season under the Mississippi climate, with oil yields and composition similar to those from other peppermint production regions. (bio-medicine.org)
  • the branch of pharmacology concerned with the effect of genetic factors on reactions to drugs. (brainscape.com)
  • COPENHAGEN, Denmark, Aug. 23 /PRNewswire-FirstCall/ -- Genmab A/S announced today that HuMax-CD4 for the treatment of Mycosis Fungoides (MF), which constitutes 75% of all Cutaneous T-cell Lymphomas (CTCL), has been designated an orphan drug by the U.S. Food and Drug Administration (FDA). (ptcommunity.com)
  • There are currently no drugs approved in the U.S. to treat AML in patients who are unfit for intensive induction chemotherapy, though hypomethylating agents are recommended by the National Comprehensive Cancer Network (NCCN) guidelines. (yahoo.com)
  • An orphan drug is a pharmaceutical agent that has been developed specifically to treat a rare medical condition, the condition itself being referred to as an orphan disease. (wikipedia.org)
  • Durhane Wong-Rieger, president and CEO of the Canadian Organization for Rare Disorders, said the decision "certainly seems to be the kiss of death" for the orphan drug framework. (nationalpost.com)
  • She is a member of the National Organization for Rare Disorders and the Drug Information Association. (xtalks.com)
  • This work could help advance efforts to develop drugs that increase levels of this enzyme in the brain, creating a chemical 'blockade' against the debilitating effects of neurodegenerative disorders," said Hui-Chen Lu, who led the study. (thirdage.com)
  • In some cases, Canadian patients can access orphan drugs not approved in Canada through Health Canada's Special Access Programme, but may have to pay for them out-of-pocket. (nationalpost.com)
  • Growing demand for customized devices combined with an expansion of technological innovation drives the major progress in personalized medicine expressed e.g. by the production of small series of individually-selected doses and tailor-made prostheses meet the anatomical needs of patients. (drugpatentwatch.com)
  • The Toscana Life Sciences incubator in Siena was a perfect fit for the project, as it provided the ideal location for small experimental trials, research and development of other drugs like Plasminogen. (europa.eu)
  • Smi 6th annual Orphan Drugs conference will discuss the controversial German pricing mechanism as well as bring together leading industry professionals to discuss the latest regulatory developments, explore how to reduce costs and learn from the latest innovations in the orphan drug landscape. (smi-online.co.uk)
  • The report focuses on global major leading industry players providing information such as company profiles, product picture and specification, capacity, production, price, cost, revenue and contact information. (sbwire.com)
  • OTCBB: ACTC), a leader in the field of regenerative medicine, announced today that it has been issued U.S. Patent Number 7736896, which is broadly directed to processes developed by ACT scientists for the production of retinal pigment epithelial (RPE) cells from human embryonic stem cells (hESCs). (pr.com)
  • The current achievements include multifunctional drug delivery systems with accelerated release characteristic, adjustable and personalized dosage forms, implants and phantoms corresponding to specific patient anatomy as well as cell-based materials for regenerative medicine. (drugpatentwatch.com)
  • With the development of the medicine proceeding, the company started looking for a new facility suitable for production. (europa.eu)