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 […]

National Institutes Of Health to Host Rare Disease Day Event; Dr. Francis Collins To Speak; Wear Jeans!

The National Institutes of Health (NIH) will celebrate the 4rd Rare Disease Day on February 28, 2011, with a day-long celebration and recognition of the various rare diseases research activities.  The event will be held in the Lipsett Amphitheater from 8:30am to 5:15pm. Dr. Francis Collins is expected to speak. A tentative agenda has been […]

Scientists at NIH, FDA & CDC To Hold Briefing On XMRV Retrovirus

Note: I plan to begin posting blogs about the XMRV retrovirus on Addi and Cassi’s website. Why would I start posting about XMRV when Addi and Cassi have Niemann Pick Type C disease, a fatal “childhood Alzheimer’s” like condition? People who are reading my blog for the first time need to know that all people […]

New FDA ‘Rare Disease’ Division Being Proposed to U.S. House Committee on Appropriations

The Kakkis Every Life Foundation is leading the effort to request $10 million in the fiscal year 2011 Ag-Rural Development-FDA Appropriations bill directing the U.S. Food and Drug Administration to establish a new review division for Biochemical and Genetic Diseases within the Center for Drug Evaluation Research (CDER), Office of New Drugs. A letter from […]

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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