LAL Deficiency (Lysosomal Acid Lipase Disease) & Wolman Disease patient support
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?
Added by Tatyana on October 20, 2017 at 2:04am — No Comments
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…Continue
Added by Brett Billmeyer on February 23, 2012 at 12:41pm — No Comments
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…Continue
Added by Brett Billmeyer on February 23, 2012 at 12:00pm — No Comments
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…Continue
Added by Brett Billmeyer on June 20, 2011 at 3:31pm — No Comments