Transcript for 'Double the Hope' for a Cystinosis Cure
When a child is born the expression. And figures in ten to. Conveys the simple gratitude. That a healthy child -- been delivered. When twins are born the number of fingers and toes doubles. As does the joy of bringing to healthy children into the world. When Patrick in general were born their parents Kevin and carries departing ten. Knew it would be challenging caring for newborn twins. Double -- crying. Double to feedings. Doubled in diapers. But within fifteen months Patrick can -- would be diagnosed with a rare terminal illness. Now. They -- -- double the courage face. An unknown future. Sister notices is a rare incurable. That affects less than 2000 children and young adults line. It's -- metabolic disease. That's slowly. And eventually. Destroys every organ in the body. Through six your child -- have a term. How do you manage and administer medication every six hours around the clock. Knowing that makes me throw up but it's the only course of action. -- you quench their insatiable thirst. When -- -- their little bodies to man two gallons of water. What do you do when your bone -- But still need to wake the children for medications. To change your sheets. At 1:30 in the morning. Departing since deal with an -- focusing as a family and I'm happy moments Sandner. Is striving for a sense of normalcy and alien life. Washington for loans and linens every morning. And becomes normal. The trip to the pharmacy for special order medications just a normal stop. -- -- -- -- And. -- nineteen doses of medication every day 365. Days a year. Is the only normal Patrick and -- unknown. From the outside. They appear healthy. On the inside. There is a battle going on in every cell of their body. -- medication help slow the. But it's not like here. -- -- -- -- -- -- -- -- Yeah. He -- from. And he. When a child is born we see the future through either -- Although great work has been accomplished by a select group of researchers who work here and funding system -- has helped other more prevalent. Like parkinson's Huntington's and -- -- liberties. Patrick and -- still need to -- Doubled them home. One thing counting the fingers and tones of their own newborn children. Is a rite of passage of real experience. -- Through your love and support. A life free -- system known since. Is within reach.
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