Orphan drug

Type: Keyphrase

Name: Orphan drug

First reported 22 hours ago - Updated 22 hours ago - 1 reports

Healthcare at Home Ltd Partners with QED Clinical Services to Deliver...

Ltd ( OrphanReach is a strategic alliance that pools the vast experience, heritage, and capabilities of both and QED Clinical Services. OrphanReach is built to support the launch of the growing number of “Rare and Orphan” products throughout the world. ... [Published PRWeb - 22 hours ago]

Entities: Clinical trials, Patient, Orphan drug, Health Care

First reported May 17 2013 - Updated May 17 2013 - 1 reports

Payer focus in the personalised world of oncology and orphan diseases

Personalised medicine is transforming disease understanding and many traditional diagnoses may be fundamentally revised based on the scientific understanding of disease mechanisms at the molecular, genetic and genomic levelsThe National Human Genome Research ... [Published PMLive - May 17 2013]

Entities: Oncology, treatments, Patient, Philadelphia Chromosome, Iressa

First reported May 17 2013 - Updated May 17 2013 - 1 reports

Antonio Tajani

European co-operation group aims to boost medicine supply

A pharma industry co-operation group promoted by the European Commission has launched a series of initiatives devoted to boosting the supply of medicines across Europe.The Platform on Access to Medicines in Europe has been created under a Process on Corporate ... [Published Manufacturing Chemist - May 17 2013]

Entities: Slovenia, Antonio Tajani, Orphan drug

First reported May 17 2013 - Updated May 17 2013 - 1 reports

CBI$s 8th Annual Rare Disease and Orphan Drug Leadership Congress

Center for Business Intelligence, 800 767 9499, www.cbinet.com ... [Published Pharmaceutical Commerce - May 17 2013]

Entities: Orphan drug, Leadership, Business Intelligence

First reported May 15 2013 - Updated May 15 2013 - 1 reports

T.PLI:good post on valuation - (repost)

Having listening to the CC, I'm out of there with a clear conclusion: The PPPS division is worth much, much, much more than the 3$ we've been talking about! :-)We already knew that CNBG will bring in about 22-24M$ gross margin. Octopharma about 15$.Let ... [Published StockHouse USA - May 15 2013]

Entities: Fruit, Orphan drug, Synergism, Medicare

First reported May 15 2013 - Updated May 15 2013 - 1 reports

FDA Food & Drug Administration

Former FDA Official Tim Cote Joins Emmaus Medical as Regulatory Advisor Emmaus Medical, Inc.

By a News Reporter-Staff News Editor at Clinical Trials Week -- Timothy Cote, M.D., MPH, former director of the Office of Orphan Product Development of the U.S. Food and Drug Administration, has joined Emmaus Medical, Inc., a specialty pharmaceutical ... [Published Pharmaceutical & Medical Packaging News - May 15 2013]

Entities: Emmaus (United States), FDA Food & Drug Administration, Orphan drug

First reported May 15 2013 - Updated May 15 2013 - 1 reports

Henry Waxman

HP&M Director Frank Sasinowski Receives NORD Lifetime Achievement Award

Earlier this week, the National Organization for Rare Disorders (“NORD”) held its annual gala here in Washington, D.C. recognizing achievements and advances in the area of rare (orphan) diseases. Of course, this year is extra special; it’s the 30th anniversary ... [Published FDA Law Blog - May 15 2013]

Entities: Orphan drug, FDA Food & Drug Administration, NATIONAL ORGANIZATION FOR RARE DISORDERS, Henry Waxman, Swedish Orphan Biovitrum AB

First reported May 15 2013 - Updated May 15 2013 - 2 reports

Orphan drug Kalydeco now available in Wales

The much-celebrated orphan drug Kalydeco (ivacaftor) will be made available to the estimated 13 eligible patients with cystic fibrosis in Wales, confirmed Mark Drakeford , Welsh Assembly Minister for Health and Social Services, last Friday (May 10 th ... [Published Terrapinn - May 15 2013]

Entities: Orphan drug, Wales, Cystic Fibrosis

First reported May 14 2013 - Updated May 15 2013 - 2 reports

Significant Near-Term Top-Line Data Catalyst And Platform Technology Should Rally Insmed

The Orphan Drug Act was passed in January 1983 to encourage companies to develop drugs that treat rare medical conditions. Companies that receive Orphan Drug Status gain certain financial incentives such as a longer period of exclusivity, enhanced patent ... [Published BioMedReports - May 14 2013]

Entities: Insmed Incorporated, Patient, infections, Pseudomonas, Cystic Fibrosis

First reported May 14 2013 - Updated May 14 2013 - 1 reports

Identifying biomarkers to articulate the value of an orphan drug: a look into Vertex’s Kalydeco case – Charles Johnson

Home » Blogs » Charles Johnson, VP of Global Medical Affairs at Vertex Pharmaceuticals, presented at World Orphan Drug Congress USA 2013 on the topic, ‘Identifying biomarkers to articulate [...]Original Article: Identifying biomarkers to articulate the ... [Published BioPortfolio - May 14 2013]

Entities: Charles Johnson, Vertex Pharmaceuticals Inc, Orphan drug

First reported May 14 2013 - Updated May 14 2013 - 1 reports

Orphan drugs

An orphan drug is defined as a medicine, vaccine or in vivo diagnostic agent that is:Medicines need to be designated as orphan drugs by the TGA before an application to register an orphan drug on the Australian Register of Therapeutic Goods (ARTG) will ... [Published Noodls - May 14 2013]

Entities: Orphan drug, Drugs, Australia, In Vivo, R.S. Corp Ltd

First reported May 13 2013 - Updated May 13 2013 - 3 reports

Nuvilex, Inc.'s Dr. Gerald Crabtree Discusses Upcoming Phase III Cancer Trials, Fast Track and Orphan Drug Status

Nuvilex, Inc. (OTCQB: NVLX) COO Dr. Gerald Crabtree is no stranger to late stage clinical trials or dealing with blockbuster therapies for cancer patients. It's likely that is exactly why he is the COO at the small Silver Spring, Maryland, biotech. The ... [Published NewsBlaze - May 13 2013]

Entities: Nuvilex Inc, Orphan drug, Phase III, INSCOR INC, Dominique Wilkins

Quotes

The National Human Genome Research Institute defines personalised medicine as "an emerging practice of medicine that uses an individual's genetic profile to guide decisions made in regard to the prevention, diagnosis, and treatment"

Payer focus in the personalised world of oncology and orphan diseases [Published PMLive - May 17 2013]

...Head of Kidney Diseases at the Ann & Robert H Lurie Children's Hospital of Chicago and the Feinberg School of Medicine of Northwestern University. "We need to make sure that physicians and patients managing cystinosis understand the risk of eye complications and are aware of the availability of an FDA-approved therapy with documented safety and effectiveness."

Sigma-Tau Pharmaceuticals launches CYSTARAN [Published Individual.com - May 16 2013]

..."We found that cancer cells need a continuous exposure at low concentrations for the drug to be of maximum effectiveness," Toretsky says. "And this strategy works extremely well in these animal models. The drug appears to be very successful."

Preclinical tests shows agent stops 'slippery' proteins from binding, causing Ewing sarcoma [Published EurekAlert! - May 15 2013]

...to benefit from treatment in these difficult-to-treat diseases," said Glenn Mattes, chief executive officer and president, Arno Therapeutics. "These data add to the growing body of knowledge indicating the potential of the compound to fill a high unmet medical need and we plan to pursue a global orphan drug designation in endometrial cancer."

Arno Therapeutics to Present Positive, New Data Supporting the Potential of Onapristone in Endome ... [Published Yahoo! Finance - May 15 2013]

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