Hi everyone!
I'm a new member here and not sure I'm writing on the right page but still I hope I can get a feed-back.
I'm from Ukraine. My son (almost 6 years old) was diagnosed CESD. 1.5 years ago we were the first in our country, at the moment there are 6 patients in total. 
Can everyone tell me in what countries Kanuma is used already for treatment? What are the results? Do all the patients receive treating?

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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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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All,

We are the parents of a CESD patient. Our oldest daughter, Maureen, was diagnosed with CESD when she was 5 years old. She is now 14 and we are glad to hear about information being shared on this website. We knew about the disease and it's complications from our daughters specialist at Childrens Hospital in Philadelphia but now can share with others in a similar situation. We also have 3 other daughters that are unaffected by the disease (they don't have it nor do they have the…

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im jessica  i lost my son in 2009 to wolmans at 4months and 20 days.. we didnt fine out he was sick till he was 2months it suck to lose a baby at 18 i didnt want that to help to me or my family is all i had no baby daddy, when my son got worse i didnt know what to do i was always in the hospital wit me till the day he passed i held him in my arms he passed in them till this day i still see him dieing in my arms..

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

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