Rare Disease Day 2012 – Spread This Call To Action To Make A Difference!

World Rare Disease Day 2012 is coming up on February 29th.  Consider these staggering statistics. Each year, 139 BILLION U.S. dollars are invested in medical research — much of it from tax payers Each year, 800,000 papers are published in medical journals by scientists Each year on average, ONLY 21 new drugs are approved by […]

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 […]

ResearchMatch.org Is Cool -The Idea, the Website and even the Logo! Is This Really From The NIH?

Great news from the NIH today — a new service called ResearchMatch.org was announced.  ResearchMatch.org is a national research study recruitment registry announced to help match volunteers with researchers who want them to participate in research studies. The registries matching model compliments Clinicaltrials.gov (a website that needs a serious facelift!) but  has one fundamental difference. […]

NIH Announces Therapeutic Trial With Supplement N-Acetyl Cysteine (NAC) To Treat Fatal Children’s Cholesterol Disease

You’ve got to LOVE supplements especially one called N-Acetyl Cysteine (NAC) when it can possibly help treat children afflicted with a rare and fatal cholesterol disease called Niemann Pick Type C (NPC). Considering there has only been only one clinical trial for Niemann Pick Type C ever conducted at the NIH (for a drug called […]

Ten Tips To Start Your Own Virtual BioTech To Find Treatments For Your Rare Disease

Ever since we went public with Addi and Cassi’s cyclodextrin treatments, I have received countless emails asking me a number of questions.  How did I find the sugar compound "cyclodextrin?"  How did I manage to get the Food and Drug Administration to approve infusions of this new compound into Addi and Cassi?  As a parent […]