Tag Archives: rare diseases

Rare Disease Day: Families and Advocacy Groups Drive Treatments for Rare Diseases

Gavin and Bella Today, we celebrate Rare Disease Day to recognize the path to treatments and cures is paved by families and caregivers working with advocacy groups, medical advisors, and dedicated researchers. Of the many people touched by and involved in the world of rare diseases, patients and their families are inarguably the most passionate advocates for rare disease research. When their disease-focused family foundations work together, pool their funds, and align their efforts, the results can be Read More >
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Orphan Drugs: Making Rare Diseases Rarer

Panel

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. 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, biopharmaceutical companies have embraced rare disease medicine and are rapidly developing the pipeline with hopes of achieving the Read More >

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Onyx CEO One of 25 Most Influential People in Biopharm Today According to FierceBiotech

Tony Coles

After a stellar year, as evidenced by inclusion in the second annual FierceBiotech “25 Most Influential People in Bipoharma Today,” N. Anthony (Tony) Coles, MD, Chairman & CEO of Onyx Pharmaceuticals sat down with Steve Usdin, Senior Editor of BioCentury for an early morning Fireside Chat at the 15th Annual BIO CEO & Investor Conference. To read more about Onyx’s stellar year and their company milestones, visit here. The biotech retained complete control in the Read More >

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FDASIA: A Step Forward for Patients

fdasia-panel

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 essential funding to the FDA by authorizing the agency to charge user fees to companies seeking to have products reviewed. Originally enacted in 1992, it must be reauthorized every 5 years. Sara Radcliffe, executive vice president of health at BIO, moderated a panel of experts and advocates Read More >

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BIO Investor Forum – Raring to Go: The Race to Treat Ultra-Rare Diseases

BIO Investor Forum

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, biopharmaceutical companies have embraced rare disease medicine and are rapidly developing the pipeline. In anticipation of an even more compelling opportunity, many companies are taking a step beyond and focusing on ultra rare diseases. Last week’s 11th Annual BIO Investor Forum in San Francisco hosted a panel, Raring Read More >

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