Research - our hope and Mael's only chance

Niemann Pick C is a very complex disease about which very little research has been done. However, a number of research projects are currently under way worldwide, and there are more and more people seeking treatments for Niemann-Pick C. There are encouraging beginnings, for example with Cyclodextrin, but a treatment that will cure the disease still appears to be a long way off, and enormous amounts of money and time are needed to make further research projects possible. Still, research is Mael’s only chance, and we are willing to do whatever we can to contribute our share. Mael is involved in an observation study for Niemann-Pick C at the National Institutes of Health (NIH) in Washington D.C., and in January 2012 we traveled for the first time with Mael and the whole family to Washington D.C., where Mael spent two days being studied. Another visit is planned for May 2013. We have high hopes for the Study on the Drug Cyclodextrin, which is also being set up at the NIH. The research team notified us by e-mail in December 2012 that they consider Mael to be a suitable candidate. We are hoping against hope that Mael can be in this study soon, and we would do almost anything to make it happen. Because at the moment this study is our best hope!

Information about Cyclodextrin

Information about the new trail by Orphazyme with the protein rhHSP70

Information about HDAC inhibition therapy

Information about Zavesca