Lou Gehrig's Disease - Motor Neuron Disease - Amyotrophic Lateral Sclerosis
Thought it had been cured by now? Still no known cause. Still no cure. Still quickly fatal. Still outrageous.

Tuesday, May 9, 2017

We Need A Toolkit

Dad was an engineer.

He taught us that you always need a good basic set of Craftsman tools, and every tool has its own use.  Never use a screwdriver when you should use an awl.  Never mess up a bolt with the wrong sized wrench.  Never hammer with anything but a hammer.  And duct tape has no correct application in plumbing.

On Friday we got the wonderful news that MT Pharma America was granted FDA approval for
Radicava (edaravone) for ALS.  There is joy in hearing that there is finally a new tool in a barren toolbox.
We can't stop building a toolkit with one new wrench of unknown size.  We need to figure out for whom Radicava works, and we need to keep complementing the toolkit with new options.  This is one tool.  We need that Craftsman set to address a nasty, heterogeneous disease like ALS.

Edaravone has been used in Japan for a number of years for stroke, and it has been approved and in use there for ALS for almost two years.  I acknowledge that this is fast approval by FDA standards, but two years is a lifetime for many with ALS.  We are grateful for Friday's news.  We must forge ahead more quickly and more aggressively.

We have a toolbox to fill with the right tools for the right people.

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