Growing Stronger organised an LP doc talk a while back and the following question was asked of Dr Wilcox, who is one of the scientists behind the BioMarin drug. I am sure I have shared this link before, but to get a full picture of how involved he exactly is, I suggest you read the April 5, 2012 blog post on this blog, sorry the direct link to the post doesn't work.
"What are your thoughts about the potential of treatment of hypochondroplasia with BioMarin's drug in trial?"
You can listen to his answer here at 7:02
I tried to transcribe as well...
"Yes the CNP should work fine on that. Biomarin isn't studying it first as there is less of them and they would need a less growth promotion, so they would probably need a lesser dose. I don't think they will be doing a trial for the drug for HCH in the near future. It may not be until the drug is approved. I wouldn't hold your breath for the next 3 years. It would take that long until it's available for hypochondroplasia.
Petite yet fierce - Ramblings of a pathologically information hungry mother about hypochondroplasia, about rearing 3 girls in a foreign country and about all the random thoughts my sleep deprived and over-stimulated brain can come up with.
No comments:
Post a Comment