race_time

This clip was taken just a few short months after Jonah was DX'd with Sanfilippo Syndrome type C- A fatal neurodegenerative disease. Jonah was just 2 years old at the time. Today he's 4.5 and still doing really well. His speech is not where it should be, but as far as one can tell he has not suffered from brain damage. Phew...
Since this clip was made we have learned that most type C children do not start to exhibit a slow down in their learning skills until the age of 6. The clock is ticking and we're in a race against time.
We have made great strides towards a cure. Jonah's Just Begun and our consortium are funding four different research projects. We hope to see a drug compound come forward in the next few years. The treatment will be the easy part. Sanfilippo Syndrome is caused by a single cell mutation, making a treatment much easier to create. However the disease is ultra rare and not considered very profitable.
Our biggest battle will be convincing a venture capitalist to help us fund an FDA approved clinical trial. Then we will have to entice a pharmaceutical company to manufacture the drug for us.
It's up to the patient advocate groups to lay all the ground work and provide the treatment's. So that VC's and Pharma can manufacture and take our treatment to market for us.
It doesn't sound fare. But the alternative to not trying... is to watch our son die a slow painful death. You can help fund a treatment, every dollar adds up. Life is a gift that keeps on giving. Go to jonahsjustbegun... to donate.
Jonny Lee Miller of the hit new television show "Elementary" is running an Ultramarathon for JJB, May 4th 2013. To sponsor go to: www.crowdrise.c...