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Today in the United States we celebrate Thanksgiving.  Canada celebrated a few weeks ago and according to Wikipedia at least 7 other countries celebrate a similar holiday at various other times of the year. This is the 149th anniversary of formal observance of Thanksgiving Day

You may be receiving two notifications about this blog post. We changed our blog subscription tool a few minutes ago.  I’ve manually copied all of the current followers to the new tool but I cannot unsubscribe you from the original tool (one of its many defects). You are the only one who can unsubscribe yourself

You all are awesome … we think there were several dozen letters sent to the FDA – just for MLD, and we know that the rare and lysosomal disease communities also contributed. Many of you have asked how you can get involved with awareness that has an impact … Every letter and phone call you