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Featured Posts (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 rare diseases with you: From the Global Genes Project.org "Rare Disease affects over 350 million people worldwide, and the largest percentage are children. There are over 7,000 rare diseases that have been identified all with very unique needs, but many with little to no support. Although each individual disease may only impact a few, together the impact is in the millions. Rare disease is not so rare." Globalgenesproject.org and rarediseaseday.org are great resources for you to find ways to raise awareness in your area. Lalsolace.org is brainstorming ways that we can raise awareness of LAL Deficiency to medical professionals, corporations, and the public throughout the year. Some of our members have asked about a LAL Deficiency walk and we are excited to explore that option. We truly need to get the attention of doctors, scientists and the FDA so that they will see the importance of approving a treatment for LAL Deficiency. We all deserve to live long, happy, healthy lives! So we will be meeting more people next week that can help lead us in the right direction to do these things. I will write about everything we have learned from the NIH and the FDA as soon as I get back. LAL Solace is going to do everything in our power to provide you with the education, resources and support you deserve! 

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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 Deficiency clinical trials. We were able to have one-on-one meetings with people who are going to be able to help us make lalsolace.org a much more educational and supportive place. We will soon be joining the National Organization of Rare Diseases and other organizations dedicated to rare diseases. We are going to be exploring the possibility of having our site translated into other languages so that this can be the global organization we need it to be. We will be working with scientists and doctors to be able to provide YOU important information about the disease, testing, clinical trails and lot of other issues. We spoke with doctors from around the world about the stories you, our members, have shared on the site about the frustrations of getting the correct diagnosis. They are listening. We are working very hard for you. Now that we have become an official non-profit organization, we will be able to outreach to more groups, join professional organizations and meet the awesome people like those at the WORLD Symposium so that we can be heard! I thought I would include a picture of our trip.

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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 http://clinicaltrials.gov/ct2/show/NCT01358370
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