Hadley Hope 10 Year Update
It was 10 years ago last week that we heard the words that forever changed our family’s lives. We can’t recall everything that was said, but certain words and phrases stand out: extremely Rare Disease, Degenerative, Terminal, there is no cure. And the bombshell: and your daughter has it too….
We were told there was nothing to do but go home and love our children, and enjoy the time we had left. Most of you know the rest of the story: That lasted only a few days before we decided to start a foundation and raise money for research to find a cure. Then, thanks to our generous supporters, and those of many other Niemann Pick Type C families, and by a true miracle: only a couple years later our kids were able to be a few of the first to receive a new experimental drug. And now that drug in finishing clinical trial, and hopefully be FDA approved within the next year. Our mission has always been to make sure ALL the NPC patients had access to the drug, and that dream is slowly becoming a reality.
The drug has been shown to significantly slow the progress of the disease, but unfortunately the drug is not the miracle cure we still need. We are reminded of this as Peyton continues to struggle with walking, talking, eating, and of course his seizures. We won’t lie, it’s been a rough year. We are also reminded almost each month, as we get another notification that an NPC patient has passed away. It seems this year has been harder, but perhaps because we knew so many of the children that passed this year.
But, as the holiday season approaches it reminds us of what we are thankful for. We are thankful that Peyton IS still able to do all of those things, even if a little less. We are thankful that Kayla is still doing well, except for some hearing loss most likely caused by the new drug, Cyclodextrin. We are hopeful that the next “cure” to come could be in the form of Gene Therapy, which we are developing right now. And, of course we are most thankful to our friends/family/supports that still reach out daily and ask about the kids.
So, we carry on. We will continue to try and find a solution to the hearing loss problem in Cyclodextrin. We will continue to support the Gene Therapy research. And, we will continue to investigate any new compounds that show promise in helping slow/stop NPC disease.