Three days from now… this coming Sunday… 11/11… Charley Turns 12 and he’s still lovin’ life!
Last year we made a huge deal about Charley’s 11th birthday. We appeared on national TV to talk about our little boy who, miraculously, turned 11 on 11/11/11. You sprung to action. And together we raised $74,000 in honor of that significant, mystical day. On that day one year ago, we vowed to deal Duchenne a devastating blow. So how did we do?
We one-upped “devastating blow” and delivered a powerful 1-2 punch.
1. Sarepta Therapeuticsshowed that kids with Duchenne who took their exon-skipping drug for 48 weeks IMPROVED on the 6-minute walk test. The boys also produced dystrophin, the protein that kids with Duchenne are missing.
Bam!
2. Summit PLC completed a phase one study in healthy adult volunteers and showed that their Duchenne medicine delivers drug levels that are expected to significantly increase utrophin production. Utrophin is a protein that can compensate for lack of dystrophin. In that study, the blood plasma concentration of healthy volunteers showed more than enough drug to increase utrophin production by 50%.
Pow!
There is a TON more to report, including extreme progress on an anti-fibrotic medication being developed by Halo Therapeutics. So keep an eye out for our annual newsletter, coming via snail mail later this month. Behind the scenes we have a tightly-run team diligently working on calisthenics and practice rounds (ie, extensive due diligence, pharmacokinetics, long-term tox studies, dosing experiments, reformulation analyses, and all the other arduous, expensive work that is required before a new medicine can be tested in humans.) This is the work that ultimately leads to major advances like the two that were reported this year.