Twas the night before Christmas, when all through the house
Not a creature was stirring, not even one mdx mouse.
250,000 stockings were hung around the world with care,
Each holding one wish about muscle repair.
The children were nestled, all snuggled in bed,
While dreams about moving danced round in their heads.
And mamma in her ‘kerchief, and I in my cap,
Could not settle our brains, worried, as we tried to nap.
When out on the lawn there arose such a clatter,
We sprang from our bed to see what was the matter.
Away to the windows we flew like a flash,
We tore open the shutters and threw up the sash.
The moon on the breast of the new-fallen snow
Gave the lustre of mid-day to the shadows below.
When, what to our wondering eyes should appear,
But sleighs full of HOPE that would lessen our fear .
With smart capable drivers, so lively and quick,
We knew in a moment, we might have to pick.
More rapid than eagles the possibilities came,
And we whistled, and shouted, and called them by name!
"Now Acceleron/Shire! Now GSK, PTC, and AVI too!
On, Ryszard! On, Hans! On, Jas! And on Stu!
To the top of the porch! To the top of the wall!
Godspeed, Godspeed, Godspeed to you all!"
We’ve waited so long, forever it seems
Trying to find hope and believing in dreams,
So thankful for research, for clinical trials
To know people like you, capable at navigating difficult miles.
We knew you would find glitches and return to the bench
Forever committed, you climbed back out of that trench.
And finding your way, plowing difficult terrain
Focused on Duchenne, we hope you always remain.
Then, in a twinkling, we heard on the roof
Several companies together thinking how to accelerate proof.
Discussing drugs for Duchenne, thinking combinations are best
To make sure all of the kids would have therapies to test
As I drew in my head, and was turning around,
Down the chimney they came all together, tumbling down.
They were dressed up all snazzy, from their head to their feet,
Talking about meetings to make sure NDAs would be complete.
A bundle of questions, they had on their back,
To ensure all new medicines are safe and on track.
Outcomes, statistics, we’re learning the lingo,
Looking forward to approvals and screaming “Bingo!”
We are thankful, we are, so thankful for you
Sometimes we don’t say it enough, it is true.
We are happy for trials, for analysis, and results
Crossing our hearts, we’ll be compliant to a fault.
All dapper and dashing, none resemble an old elf,
And we laughed when we saw them, in spite of ourself!
A wink of their eyes and a twist of their heads,
Soon gave us to know we had nothing to dread.
They spoke nary a word, sticking straight to their work,
Which is scary at times, but when they turned with a jerk,
They smiled about trials now online, and recruiting,
As they gave a kind nod, I was already computing!
Springing back to their sleighs, they gave a loud whistle,
And away they all flew like the down of a thistle.
In ONE VOICE they exclaimed, as they drove out of sight,
"Have HOPE all of you, and to all a good-night!"