Parents To Seek FDA Orphan Drug Designation For Cyclodextrin To Treat Rare and Fatal Cholesterol Disease

On Tuesday morning at 11am, Addi and Cassi are going to undergo volumetric CT scans of their livers and spleens.  The tests are being conducted by doctors to determine if the cyclodextrin IV infusion treatment the twins are receiving under compassionate use INDs approved by the FDA are reducing the size of their enlarged cholesterol […]

How Medical Decisions By Doctors Are Leaving Families In Financial Ruin

This is a medical story that is happening all over America – a story about doctors making medical decisions and not listening to patients (in this case an astute Mom).  This is a story that is all too familiar to Niemann Pick Type C families  — misdiagnosis for months or even years when symptoms seem […]