Gene Therapy

Thought we would pass on some good news in the field of NPC Research - Many people have asked us if the Cyclodextrin Drug that the kids are on is a cure.  It is not.  But, it IS significantly slowing the progress of the disease, which gives us time and hope of finding another solution, which the Hadley Hope Fund continues to support financially and administratively.

The following article was released last week regarding the potential for Gene Therapy in treating NPC.  This is something we have been supporting and monitoring, and the early test results have been released, showing promise in the mouse model.  Gene therapy in NPC is still a few years away from even being thought of as a treatment, but it is being used in a handful of other diseases already, and being testing in many more.  These early results are exciting.

Keep the good thoughts and prayers coming, and together we'll continue the fight!  With the end of the Cyclodextrin clinical trial in sight, and promising news in other studies, we have reason for hope!

Read the article from the NIH here: