How do you find a cure?

FARA's mission is to develop treatments, and ultimately a cure, for Friedreich's ataxia.  Where would you start?

Understanding the exact cause of FA, and the cellular disfunctions that lead to symptoms in FA patients, was a huge breakthrough!  FARA is currently supporting a range of work aimed at treating each specific mechanism of damage.  FA researchers believe that treatment will ultimately come in the form of a "cocktail" therapy - that multiple therapies aimed at different sources of damage can be used in conjunction with one another to successfully treat FA.  The chart below displays the different treatment approaches current being explored with FARA's support. (Click on the chart to make it bigger.)

FARA research pipeline as of February 1, 2011

The treatment pipeline depicts the progress of research and development in leading therapeutic candidates. On the horizontal axis, therapies are grouped based on their means of action (where/how they might work in the cell), the technological approach, or the problem being addressed. The vertical axis shows the current stage in the development process. The first two stages, research and pre-clinical, take place in the lab and represent early discovery and development. The stages “Phase 1” through “available to patients” are clinical trials or studies.  

Garrett can no longer participate in a clinical trials because his advanced symptoms make it impossible to measure a treatment effect on standard metrics like balance or hand coordination.  But he is currently one of only 20 people to receive an experimental, pre-clinical drug aimed at treating mitochondrial disease.  Since FA is a rare disorder, it's important that FA patients participate in the search for a cure.  Garrett will be a part of that achievement, when the day comes.  And we can be too by making financial contributions to FARA that allow them to support this effort.

Garrett in 2005, a better year for the Eagles

Final Contest Reminder!

You still have 2 days to enter a prediction in the Jenny's Light 10K contest!  Just leave a comment back over here with your best guess at my finishing time.

And thanks!

In other news this week, I'm another year older. And hopefully wiser and faster! Thanks for all the nice birthday wishes. They made for a great day!

My cube on Wednesday morning!

More cube decorations arrived.  I do love flowers!

It takes courage to grow up and become who you really are.
~ e.e. cummings