Blogs about: Orphan Drugs
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Rare Diseases: Upcoming Documentaries (06.19.13)
This is a third in a series on rare diseases and the movies. This Blog Post reviews several upcoming scheduled documentaries. I – “The Life We Live” Ambry Genetics, located in Aliso Viejo, California,… more →
Rare Diseases: Upcoming Documentaries (06.19.13)
wrote 5 hours ago: This is a third in a series on rare diseases and the movies. This Blog Post reviews several upcoming … more →
Orphan Drugs And Rare Diseases : New Start-up And IPOs (06.18.13)
wrote 1 day ago: This Blog Post discusses a new startup and Initial Public Offerings (IPOs) in the rare disease and o … more →
DMD Race For First FDA Approved Drug: Sarepta Therapeutics Business Developments
wrote 2 days ago: This is the third in a three part series on recent business developments for the three competitors a … more →
Apremilast: Celgene’s Orphan Drug Phase II Results For Behçet’s Disease
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wrote 4 days ago: Celgene’s orphan drug Apremilast is an oral agent that is currently under clinical development … more →
Rare Diseases: Case Study Of A Patient Advocacy Group And Gene Therapy
wrote 5 days ago: The influence of patient advocacy groups on developing new treatments for rare diseases is powerful. … more →
Orphan Drug pathway to market
wrote 6 days ago: Source: PMLive How an orphan drug eventually ends up as a treatment for patients This infographic ex … more →
Orphan Drugs And Rare Diseases: Global News (06.12.13)
wrote 1 week ago: Recent orphan drug and rare disease global developments are in the news. Here is a list and descript … more →
Celgene’s Orphan Drug Revlimid Receives FDA Approval
wrote 1 week ago: Celgene announces on June 5, 2013 (scheduled FDA PDUFA date), that the FDA approves the supplemental … more →
Rare Diseases And Orphan Drugs: New Startup And Other Happenings (06.05.13)
wrote 1 week ago: The following Blog Post discusses a new startup and other happenings in the rare disease and orphan … more →
Orphan Drugs: Global Regulatory Events (06.04.13)
wrote 2 weeks ago: Recent orphan drug global regulatory events are in the news. Here is a list and description of recen … more →
CBI’s Orphan Drug Innovation Summit
wrote 2 weeks ago: Have you registered for CBI’s Orphan Drug Innovation Summit yet? Join me at this highly interactive … more →
FDA May 2013 Products Receiving Orphan Designation
wrote 2 weeks ago: The chart below identifies the FDA May 2013 Products Receiving Orphan Designation. The chart is gene … more →
Rare Diseases: Pediatric Priority Review Voucher Incentive Program (05.30.13)
wrote 2 weeks ago: Special Note: By request, the March 2013 Blog Post is republished with updates. The FDA Safety & … more →
GlaxoSmithKline: FDA Approval Of Two Orphan Drugs Targeting Metastatic Melanoma
wrote 2 weeks ago: GlaxoSmithKline (GSK) receives FDA approval on May 29, 2013, for the following two orphan drugs for … more →
Orphan Drugs: The Rising Stars
wrote 3 weeks ago: Thomson Reuters publishes a May 2013 report, “The Ones to Watch”, that identifies and analyzes drugs … more →
Making the Case for Paying the High Cost of Orphan Drugs
wrote 3 weeks ago: What exactly is an orphan drug, and why are they so expensive? According to the FDA, orphan drug sta … more →
Orphan Drugs And Rare Diseases: Global News (05.28.13)
wrote 3 weeks ago: Recent orphan drug and rare disease global developments are in the news. Here is a list and descript … more →
Orphan Drugs: EMA’s COMP May 2013 Meeting Results
wrote 3 weeks ago: EMA’s Committee for Orphan Medicinal Products (COMP) held a meeting May 14 – 15, 2013. The EMA COMP … more →
Rare Diseases And Foundations: Gene Therapy Developments #3
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wrote 3 weeks ago: This is a third in a series of Blog Posts this week, on the topic of gene therapy developments in th … more →
