A True Healthcare Reform Idea – Priority Review Vouchers For Ultra Rare Diseases

This week I have been reading all about priority review vouchers. Henry Grabowski and other faculty at Duke University came up with the idea for providing an incentive for drugmakers to target certain tropical and infectious diseases (such as TB, malaria, cholera, dengue, leprosy) affecting poor nations.  Senators Sam Brownback (R-KS) and Sherrod Brown (D-OH) […]

Numbers Don’t Add Up – Only 347 New Drugs Approved By FDA For 30 Million Americans With Rare Disease in Past 25 Years?

When it comes to finding Rare Disease statistics, the same old statistics and facts keep popping up again and again. I realize data is difficult to gather on 7000 different rare diseases that afflict 30 million Americans but it’s frustrating that sports teams and Wall Street have more comprehensive statistics when millions of people across […]

The Road to Building a New Orphan Drug – Am I Mad?

On February 25-26, 2010, I will be attending the inaugural FDA Build-an-Orphan-Drug-Workshop hosted by the Center for Rare Disease Therapies at Keck Graduate Institute (KGI) in Claremont, Calif.  FDA agency experts will be on hand to provide guidance on applying for orphan drug designation. I will be attending the workshop with Dr. Ron Browne, hired […]

American Heart Association Features Addi and Cassi’s Fatal Genetic Cholesterol Disorder

Thank you to the American Heart Association for featuring Addi and Cassi Hempel’s story and increasing awareness of Niemann Pick Type C, an ultra rare and fatal cellular cholesterol disease that is trying to steal our twins away from us. We hope that by reading our story, more people will understand the significance of the Niemann Pick Type C […]

Family of Twins with Rare and Fatal Cholesterol Disease To Seek FDA Approval To Deliver Cyclodextrin Into the Brain

The tagline of the new movie, Extraordinary Measures, about John Crowley’s fight to save his children from the rare and fatal Pompe disease is….Don’t Hope For a Miracle – Make One! We are now working on making our own miracle  – a new compassionate use investigational new drug (IND) protocol and application that we plan […]

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