Alzheimer’s Patients Presenilin 1 Gene Defect Causes A Lysosomal Storage Disorder

A research paper by Dr. Ralph Nixon, director of the Center of Excellence on Brain Aging and the Silberstein Alzheimer’s Institute at NYU Langone Medical Center, was published today in the journal Cell reporting that Alzheimer’s patients with genetic mutations in the presenilin 1 gene have disruptions in the cellular protein recycling process mechanisms in […]

Orphan Drug Act – A Collosal Failure Considering Rare Disease Drug Statistics

For the past week, I have been obsessing over rare disease statistics ever since it was published that 80% of all rare diseases are ‘ultra orphans’ – affecting 6,000 people per disease state or less – and only 15% of all rare disease drug applications filed with the FDA are for ‘ultra orphans.’ Yesterday, I […]

FDA Grants Orphan Drug Status For Cyclodextrin Compound To Treat Fatal Genetic Cholesterol Disease

Drum roll, please! Dr. Caroline Hastings at Children’s Hospital Oakland and Research Center received a call on Friday afternoon from the U.S. Food and Drug Administration regarding the orphan drug submission we made at the end of February. While we have not received the official letter by mail (probably next week), it appears our Orphan […]

Guidelines To Write and Submit an Orphan Drug Application For A Rare Disease

While working on our orphan drug application for Cyclodextrin for the treatment of Niemann Pick Type C disease, I searched the Internet looking for examples of an actual orphan drug application that was filed with the U.S. Food and Drug Administration. Not surprisingly, I could not find any ‘real applications’ that could be used as […]

FDAs Orphan Drug Workshop Featured in Wall Street Journal – Push To Cure Rare Diseases

Below is a story that ran in today’s Wall Street Journal (Page A 3) on the FDA workshop I attended a few week ago at the Keck Graduate Institute in Claremont, CA.  We filed an orphan drug application for Cyclodextrin for the treatment of Niemann Pick Type C disease, an ultra rare cholesterol disease that […]

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