Wendy and I went to see the movie this afternoon. I am very torn. On one hand I am very grateful to the big studios for making a rare disease movie. Particularly a lysosomal storage disorder like Pompe disease which is very similar to Batten Disease. On the other hand, they have to make it fit into a two hour time frame so they really can’t delve into all the emotions, ups and downs, difficulties etc of a life outside the norm caring for affected children. They made “the special medicine” look so easy and it’s not. They didn’t show how hard the research labs work, they didn’t show how difficult it is to get funding, they didn’t show the countless hours, time and money that families dedicate to fundraising and raising awareness about their rare disease. Many times these families don’t have any of these things to spare but we do it anyway. I really, really, really appreciate the publicity and exposure of rare diseases but it is never enough as long as there are kids dying out there.
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