A fellow DMD parent posted this today and it expresses so well how we are all feeling about our wait for the PTC-124 trial to continue!
Once upon a time, in a land called Nooooo Joisey there was a sound wrenching tearing blast of a sound;
At first, like the cry of a cat who accidentally got caught in the rinse cycle of a Maytag washer; the sound rising louder and higher, more shrill, becoming as a tortured human voice exclaiming wonder, and surprise. And lo, the sound called PTC-124 was heard throughout the land. An a great silent anticipation was born unto the people. The “Great Waiting” had begun.
Three years later, 2004 another, screeching, agonizingly, heart wrenching tore through the night sky, incoherently proclaiming “very soooooon!”, “veeeerrrryyy sssoooooooon!!!”. A study has been done; many words and deeds were proclaimed and heralded throughout the communications media; The Great Waiting continued, anticipation renewed, the time of promise was at hand.
Then silence, for a moment all was calm; a slow deep rumbling was felt deep in the earth, growing louder and more ominous, a year later a quick flash of a study was done. The Great Waiting was becoming confused.
Another year and the beast once more tore at the ears and heart strings , crying in a shrill seductive voice, “Soooooooooo”, “Verrrry sooooooon”; The sound returned to the depths from whence it came, sinking slowly drowning the hopes of those it had awakened from their fear. The Great Waiting, was punctuated with the Great Frustration of Obfuscation, was the voice friend or foe, was the promise truly real. On the other side of the universe, those who gasp for air were treated to the magic and wonder as the great PTC-124 bore them from the depths to heights undreamed in the days of the ancients. Alas, it was a universe away.
The rumbling subsided, but occasionally a staccato of outbursts, a cacophony of garbled inhumanly giddy voices spewed out on the land the promises of old to those who may walk. Now the Great Waiting has become the Great Yearning, seeking to know the truth.
But the earth trembles again now, slowly in abrupt shakes and violent quivers, building slowly into a creaking crescendo convulsing thrashing to break free and once again.
After so many years, I begin to fear and loath the silky seductive voice of promise, the beast that tears out the heart and soul, tempting and swelling the breast with promise only to slink away to it’s hole leaving nothing but desperation and futility in it’s wake.
A cry in the night wind, heard faintly receives no answer; PTC-124 where are you, have you forgotten about us….
So anyway, Qtr-1 of 2008 is gone in 4 days, as Yoda would Say;
Promise on paper it was, made known details were, fulfill them you must……
Coping – and Continuing to Cope with the knowledge that a drug that works that has proven its efficacy is not being released to those who need it.
MikeP., one can only cope so long before the tipping point is reached.
