BIO Investor Forum – Raring to Go: The Race to Treat Ultra-Rare Diseases
The signing of FDASIA by President Obama in July reinforces the country’s commitment to fostering innovation for rare diseases. With a record 460 medicines in late-stage clinical trials,...
View ArticleFDASIA: A Step Forward for Patients
Earlier this year, Congress passed and President Obama signed the FDA Safety and Innovation Act (FDASIA) which included a re-authorization of the Prescription Drug User Fee Act (PDUFA). PDUFA provides...
View ArticleOrphan Drugs: Making Rare Diseases Rarer
A panel at the BIO CEO & Investor Conference provided an analysis of the thriving orphan drug market opportunity featuring industry, clinical and regulatory experts specializing in rare diseases....
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Prosensa’s IPO and the Value of the BIO Investor Forum
Prosensa (NASDAQ: RNA) is a Dutch biotechnology company engaged in the discovery and development of RNA-modulating therapeutics for the treatment of genetic disorders. Our primary focus is on rare...
View ArticleFDA Check-Up: Drug Development and Manufacturing Challenges
I have over 27 years of experience in the biotechnology and pharmaceutical industry. My current company, OncoMed Pharmaceuticals, is working at the cutting edge of oncology research, focused on...
View ArticleForbes: Faster FDA Approvals Have Not Caused More Drug Safety Problems
In 1992, Congress, industry, and the FDA worked together to create the Prescription Drug User Fee Act (PDUFA). This program ensures that FDA has the ability to hire additional reviewers to expedite the...
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Mito What???
Mitochondrial Disease: The Most Common Disease You’ve Probably Never Heard of… About 20 years ago, mitochondrial disease was virtually unheard of. Just 10 years ago, it was considered rare. Today,...
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