FDA – Page 5 – Patient Worthy

Is This New MDS Treatment Worth Getting Excited About?

Post author:Kiki Jones
Post published:June 17, 2016
Post category:Myelodysplastic syndromes Rare Disease

In the rare disease world, nothing causes rippling waves of excitement quite like a new drug announcement. So prepare for a tsunami, because that’s exactly what you’re getting… sort of. Spring…

Breakthrough! Why You Should Care About FDA HAE Designations

Post author:James Ernest Cassady
Post published:October 26, 2015
Post category:HAE Rare Disease

Recently, the FDA gave "breakthrough therapy" status to a potential new drug that may help prevent HAE attacks. But how does that affect you, the patient? What does "breakthrough therapy" really mean?…

The FDA Just Gained Big Brownie Points from Rare Disease Patients

Post author:Winnie Nash
Post published:October 22, 2015
Post category:Rare Disease

Earlier this year, the FDA took bold steps, launching them closer to efficient rare disease drug development. And now, the potential improvement for rare disease patients could be huge. Why?…

Is the New FH Drug Inspired by Star Wars? Who Cares, It Works

Post author:Patient Worthy Contributor
Post published:July 22, 2015
Post category:Familial Hypercholesterolemia Homozygous Familial Hypercholesterolemia Rare Disease

Standing in front of a crowd and talking about your HoFH can be intimidating. Imagine if that crowd was a panel of doctors and scientists with the US Food and…

Is This New MDS Treatment Worth Getting Excited About?

Breakthrough! Why You Should Care About FDA HAE Designations

The FDA Just Gained Big Brownie Points from Rare Disease Patients

Is the New FH Drug Inspired by Star Wars? Who Cares, It Works

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