Children with a form of Alzheimer’s

Twin girls named Addi and Cassi from Reno Nevada just started a new medication called cyclodextrin for a disease they both have and are the only ones in the country that are being treated for their condition. The five year old twins have a very rare disease called Niemann-Pick Type C disease or also known and NP-C. This disease is like a form of Alzheimer’s but targets children. They progress and then hit a certain point in their lives and begin to regress losing ability to talk or control over bowl movements. The medication cyclodextrin is not FDA approved but was allowed by the FDA to be used in the hopes that it could possibly help the two children. The FDA doesn’t normally let people use medication that isn’t approved unless they are at a point that there aren’t any other options or alternatives that will help them. In Addi and Cassi’s case at the moment there isn’t a cure or FDA approved medications for their condition.I think that it is so unfortunate that these girls have to go through this. I know how hard it was when my grandfather went through Alzheimer’s let alone five year old girls. At least my grandfather had lived a full filling life. I really hope that this medication helps them so they are able to talk to their parents again!!



http://www.comcast.net/articles/news-health/20090419/Sick.Twins.Treatment/

I have never heard of this. This is very surprising since that they are only five years old. I also hope that the medications would work. I agree with you on that the elderly at least got to live a full filling life I just hope that the medicine will help them. I know a few people that have Alzheimer’s and it’s horrible what they go through and what their families go through.

How very sad this is! I see that the FDA approved it being used through the "compassionate use" program. I am glad that they decided it would be allowable. I did notice that the program is only starting the medication at half the recommended dose at this time because the FDA needs to monitor these girls every couple of weeks. Hopefully the girls will start to improve and have no bad side effects or allergic reactions. In a best case scenario, this could be a god-send to everyone because normally the testing that the FDA puts drugs through can take years before humans are allowed to be tested. If they are successful with these two girls, they may well save many lives.

Good luck to them, their family and the medical team involved in their care.