From The Valley of Death to The Valley of Cures – NIH TRND Program Supports Multiple Rare Diseases

Thank you Dr. Francis Collins, Dr. Chris Austin, Dr. John McKew and others at the NIH who are all involved in driving the National Institutes of Health’s Therapeutics for Rare and Neglected Diseases (TRND) program forward. TRND has been a huge success since launching five pilot projects last year, including one program that focuses on […]

Why Vote4Hope? How Kickstarting Cures For Rare Disease Through The Pepsi Refresh Project Can Help Kids

Last week, I thought about trying to raise $250k in the Pepsi Refresh Project for The Addi and Cassi Fund and for Niemann Pick Type C disease research but being part of something larger that can make a difference for millions of ‘rare kids’ is important to me. That’s why I decided during the entire […]

Cystic Fibrosis and Niemann Pick Type C Linked Through Cholesterol Abnormalities and cAMP Cell Signaling Pathway

A few months ago, I wrote a blog about the connection points between Duchenne Muscular Dystrophy (DMD) and Niemann Pick Type C (NPC) and how more research dollars are needed for collaborative research projects between the two deadly diseases. Today, I am writing about the connections between Cystic Fibrosis (CF) and Niemann Pick Type C. […]

Calling Jerry Lewis – Scientists Report Understanding Niemann Pick Type C Could Lead To Treatments For Duchenne Muscular Dystrophy

There are new reasons for hope not only for families of Niemann Pick Type C kids but for families with children suffering from Duchenne muscular dystrophy, a fatal recessive X-linked form of muscular dystrophy that strikes 1 in 3500 boys and leads to paralysis and ultimately death. According to a research paper released from the […]