Mary Pruitt's Blog (3)

Rare Disease Day...Getting the Word Out!

Next week, on February 29 it will be Rare Disease Day! A day to recognize those of us who have been touched by a rare disease. And with LAL Deficiency, we certainly quality for a rare disease! Our Board will be traveling to Washington D.C. for Rare Disease Day events. There will be a day for patient groups at the National Institute of Health and a day at the FDA to discuss what has been going on in terms of research and treatments for rare diseases. I just wanted to share some facts about…

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Added by Mary Pruitt on February 23, 2012 at 12:41pm — No Comments

WORLD Symposium 2012

Two weeks ago, Stephanie Dykes, Dakota's mom and our Board's secretary, and I travelled to San Diego for the WORLD Symposium. This was a conference dedicated to Lysosomal Diseases, such as Wolman's Disease and CESD. It was a gathering of medical professionals, scientists and patient advocacy organizations, such as LAL Solace. Stephanie and I were able to meet so many wonderful people who are working to raise awareness and treat these diseases. We listened to speakers discussing the LAL…

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Added by Mary Pruitt on February 23, 2012 at 12:00pm — No Comments

Study Opportunity

Synageva is currently studying the natural history of early onset (Wolman Disease) LAL Deficiency.  The information from this study is very important to the understanding of Wolman disease since it is so rare and there is no information summarized from large groups of patients available in the literature.  If you are interested in having your child’s data included in this study, please contact one of the active clinical study sites or the sponsor representative listed on … Continue

Added by Mary Pruitt on June 20, 2011 at 3:31pm — No Comments

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