Take the Holland family of Fort Worth, Texas. Steve and Amy Holland had big dreams for their three kids. Now they just wish for three healthy children.
CBS News Medical Correspondent Elizabeth Kaledin reports all three of their childrenSpencer, Madison and Lanieare dying.
They have a genetic disorder called Mucopolysacharidosis, or MPS. They lack an enzyme needed to break down a protein crucial to normal growth. As a result the protein builds up in joints and organs, generally causing death by the age of ten.
The Holland children were diagnosed within a week of each other.
"I thought about Maddie and Lanie in ballet classes, and Maddie and Lanie walking down the aisle, Spencer going to the prom and driving a carit was as if all of it had been taken away from us," says Amy Holland.
But little did the Hollands know that an even more agonizing scenario was still to come.
In 1997, Spencer was one of ten children enrolled in a clinical trial of an experimental treatment in which the missing enzyme is replaced. At the time he was going downhill fast.
But within weeks of treatment, Amy Holland says, "I could just not believe the difference in my child and that was the day that I realized it was working."
The Hollands expected results from the trial would win the drug swift approval by the Food and Drug Administration, but that didn't happen.
"Looking at a drug in ten patients establishes safety in those ten patients. We still don't know enough about the product," says Dr. Marlene Haffner of the FDA.
Now the FDA wants a second trial, meaning Madison and Lanie wait and weaken, while their brother -- who still gets the drug -- thrives. "We feel like absent doing something radical like stealing the enzyme or something, I don't know what else I can do," Steve Holland says.
"The longer they take to make up their minds on this drug, the more my child suffers," says Amy Holland.
But no matter how heartbreaking the story of need, it's not so simple for officials at the FDA.
"I hope you really know how hard we try to get these drugs out to patients, but the last thing you want is to find out that a drug is hurting people," says Dr. Haffner.
Spencer's doctor, Lewis Waber, says the drug is safe and he believes more data could be collected while sick children are taking it.
"What needs to be fixed here to give these children a shot at survivalI think the FDA needs to loosen up their requirements for these very rare diseases."
That said, the waiting continues for the Hollands. Theyre hoping to get both girls into the next clinical trial-- which has been delayed. If the girls don't qualify, their only hope is to apply to the FDA for compassionate use of the drug.
"Every single thing we do as a family takes on a deeper meaning and we cherish every single task and every single accomplishment that our children have," says Amy Holland.
And for this family, not a day, not a minute can be taken for granted.