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
- Most up-to-date information about the planned Cyclodextrin study at the National Institutes of Health
- Description of the Cyclodextrin-Study: December 2012
- Article of the Cyclodextrin-Study: February 2013
- Informationen about Cyclodextrin from a family-website (USA:) August 2012
- Video about Cyclodextrin use (USA): 2011
- Video about success with Cyclodextrin (USA): 2010
Information about the new trail by Orphazyme with the protein rhHSP70
- PowerPoint presentation: April 2013
- Summary by www.niemannpick.org.uk: April 2013
- Information about Orphan Designation in EU: February 2013
Information about HDAC inhibition therapy
- Article about HDAC therapy from the University of Notre Dame: November 2012
- HDAC on addiandcassi.com: March 2011
Information about Zavesca
- Information about Zavesca from the manufacturer Actelion: 2009
- One-Year Results On The Use Of Miglustat (Zavesca®) in Niemann Pick Type C: 2007