Let me tell you a little story about a sailing ship, on the high seas of the northern Pacific...
Once upon a time, a ship was sailing across the great ocean, a storm came out of nowhere, and after lashing the ship with wind and waves for what seemed hours on end, it swept one great wave across the deck of the ship, and faded away.
It left in its wake a tangled mess of ripped sails and ropes - the ship's dory and life rafts torn and scattered across the waves of the sea, swept across the horizon. All on board were spared from injury but one. One small boy was swept away from his family, and off the deck by the last wave, and tossed into the cold unfeeling hands of the Pacific Ocean.
He cried for help, and immediately the crew spotted his head - bobbing up and down with the waves. Terror in his eyes as he struggled to stay afloat. In the cold Pacific, it takes but 20 minutes of exposure to be fatal. After only 10-13 minutes - unconsciousness sets in - and rescue becomes more and more difficult. The clock was ticking.
The crew were first rate sailors, and immediately the ship set course to rescue the boy overboard. Immediately the captain noticed the lifesaving gear the storm had ripped away, and formed two task forces.
To the first, he assigned the task of replacing the dory. A makeshift raft, and the means to launch it from the storm torn decks high above the sea. This was to be the replacement lifeboat - for this rescue, as well as those to come in the days ahead, as the storm threatened it's return. The crew worked furiously lashing together planks and rigging pulleys and tackle. They were making progress by leaps and bounds - but their solution was going to be an hour or two away. Far too late for the boy in the ocean. Still, they threw all they had into their task. Heart, mind, and soul.
To the second task force, the captain assigned the task of doing whatever they could to keep the boy alive until the liferaft was ready. Working with the material they had at hand, they fashioned together a makeshift life preserver out of old clothes and hastily filled hot water bottles. It wasn't pretty, but it did seem to offer some buoyancy and warmth, and a mere 9 minutes after the boy was swept over, the crew managed to toss the ramshackle device near enough to the boy for him to cling to. All eyes were glued to the boy - would it be enough? The minutes began to tick by. First one, then 2 minutes passed. The boy was still afloat - now in the water for over 10 minutes. It was sometimes hard to tell, but he was not fading as fast as before. But his head still bobbed in the water - would it be enough?
Suddenly orders were shouted, and the ramshackle clump of hot water bottles and old clothes were pulled away. Pulled back up to the ship so they could be examined to see how well they were working. Alone again on the waters the boy began to struggle. Cold waves lapped over his head and his strokes were beginning to weaken. Clearly he was doing worse now, back on his own, than he was with the device. The task force argued amongst themselves about whether or not it was working, only to be called up short by the anguished wail of the boys family aboard the ship. "Have mercy!" they cried - begging the crew to redeploy the device, beseeching them to give the boy a chance. Minute after minute passed, as the crew wrestled with whether to re-deploy the only chance the boy had of surviving until the raft was ready. The decision to throw it out to him again was made - but by this time, the exhausted boy was nearing the point of unconsciousness. The boy grabbed back on, but by this time the cold sea had stolen the strength in his arms, and his leaden feet were not strong enough to hold on. And he began to slip away...
Duchenne Muscular Dystrophy steals the ability to walk from most of its victims between the ages of 10 and 13 years old. Doug was on a promising new drug from the age of 9 to 11 - and we felt it showed a noticeable improvement in his daily living strength and endurance. The problem is that measurement of results of treatments on this disease are difficult. The drug didn't meet the manufacturers hopes, so they stopped the study to "analyze the results". It has been a year now of "analysis" and despite the crying out of parents, promises of re dosing are still months away. Today is my son's 12th birthday. Our kids are slipping beneath the waves. We can see it each and every day.
Please urge researchers and regulators alike that unnecessary delay and deliberation is not just delaying the treatment and cure - it's costing us our children.
- an anguished parent.
Once upon a time, a ship was sailing across the great ocean, a storm came out of nowhere, and after lashing the ship with wind and waves for what seemed hours on end, it swept one great wave across the deck of the ship, and faded away.
It left in its wake a tangled mess of ripped sails and ropes - the ship's dory and life rafts torn and scattered across the waves of the sea, swept across the horizon. All on board were spared from injury but one. One small boy was swept away from his family, and off the deck by the last wave, and tossed into the cold unfeeling hands of the Pacific Ocean.
He cried for help, and immediately the crew spotted his head - bobbing up and down with the waves. Terror in his eyes as he struggled to stay afloat. In the cold Pacific, it takes but 20 minutes of exposure to be fatal. After only 10-13 minutes - unconsciousness sets in - and rescue becomes more and more difficult. The clock was ticking.
The crew were first rate sailors, and immediately the ship set course to rescue the boy overboard. Immediately the captain noticed the lifesaving gear the storm had ripped away, and formed two task forces.
To the first, he assigned the task of replacing the dory. A makeshift raft, and the means to launch it from the storm torn decks high above the sea. This was to be the replacement lifeboat - for this rescue, as well as those to come in the days ahead, as the storm threatened it's return. The crew worked furiously lashing together planks and rigging pulleys and tackle. They were making progress by leaps and bounds - but their solution was going to be an hour or two away. Far too late for the boy in the ocean. Still, they threw all they had into their task. Heart, mind, and soul.
To the second task force, the captain assigned the task of doing whatever they could to keep the boy alive until the liferaft was ready. Working with the material they had at hand, they fashioned together a makeshift life preserver out of old clothes and hastily filled hot water bottles. It wasn't pretty, but it did seem to offer some buoyancy and warmth, and a mere 9 minutes after the boy was swept over, the crew managed to toss the ramshackle device near enough to the boy for him to cling to. All eyes were glued to the boy - would it be enough? The minutes began to tick by. First one, then 2 minutes passed. The boy was still afloat - now in the water for over 10 minutes. It was sometimes hard to tell, but he was not fading as fast as before. But his head still bobbed in the water - would it be enough?
Suddenly orders were shouted, and the ramshackle clump of hot water bottles and old clothes were pulled away. Pulled back up to the ship so they could be examined to see how well they were working. Alone again on the waters the boy began to struggle. Cold waves lapped over his head and his strokes were beginning to weaken. Clearly he was doing worse now, back on his own, than he was with the device. The task force argued amongst themselves about whether or not it was working, only to be called up short by the anguished wail of the boys family aboard the ship. "Have mercy!" they cried - begging the crew to redeploy the device, beseeching them to give the boy a chance. Minute after minute passed, as the crew wrestled with whether to re-deploy the only chance the boy had of surviving until the raft was ready. The decision to throw it out to him again was made - but by this time, the exhausted boy was nearing the point of unconsciousness. The boy grabbed back on, but by this time the cold sea had stolen the strength in his arms, and his leaden feet were not strong enough to hold on. And he began to slip away...
Duchenne Muscular Dystrophy steals the ability to walk from most of its victims between the ages of 10 and 13 years old. Doug was on a promising new drug from the age of 9 to 11 - and we felt it showed a noticeable improvement in his daily living strength and endurance. The problem is that measurement of results of treatments on this disease are difficult. The drug didn't meet the manufacturers hopes, so they stopped the study to "analyze the results". It has been a year now of "analysis" and despite the crying out of parents, promises of re dosing are still months away. Today is my son's 12th birthday. Our kids are slipping beneath the waves. We can see it each and every day.
Please urge researchers and regulators alike that unnecessary delay and deliberation is not just delaying the treatment and cure - it's costing us our children.
- an anguished parent.
