Who is Robert?
Robert is a fun-loving and super sweet 6-year-old little boy who has never met a stranger. Ask anybody who knows Robert, and they will beam describing his unique personality, total disregard for his ailments that disrupt his everyday life, and inspirational character. Despite his larger-than-life personality, since being diagnosed over 4 years ago with Multipolysacharides IVA (Morquio Syndrome), Robert has grown less than 2 inches per year and the growth is declining.
The Severe Effects of Morquio Syndrome
This disease is a rare genetic disease that prohibits the body from breaking down certain sugars. It’s severely progressive, and the effects will get significantly worse with time.
Just a decade ago life expectancy for a child with Morquio would be about 20 years old and while recent medical advances should help to extend Robert’s life expectancy, the effects of the disease are real and apparent. Robert has already gone from a very coordinated 2-year-old in the 25th growth percentile to a 6-year-old who, simply put, cannot keep up with peers, fights to climb a flight of stairs, and does not register on the growth chart.
While most children Robert’s age play carefree and actively participate in sports, Robert has, with rare complaint:
- Had a port surgically implanted in his chest 4 years ago,
- Undergone over 200 weekly 6 hour enzyme replacement treatments (that have limited known effects on anything but assisting energy levels),
- Spent 6 months in a neck brace in 2020/2021 while 4 vertebrae were surgically fused in an effort to protect his spine from severing his spinal cord; and
- Had major hip and leg reconstruction in which new hips were created from grafting, pins were inserted in his ankles, and screws were put in his knees, all of which required 6 weeks in a body cast and continuous physical therapy to re-learn how to walk.
Further, this disease does not stop with effects to the bone. Robert will suffer (and in some instances, has already suffered) from numerous other health defects to his “soft tissues”, including life-threatening heart, lung, and spinal cord issues, together with hearing and eyesight loss, compromised immunity and low energy.
Robert’s body is failing him every day, getting worse right in front of our eyes, and he will have limits on his quality of life and quite likely, a more limited life expectancy.
We live in unparalleled times where real medical advances not only can happen, but with the significant amounts of financial assistance are actually happening. We have talked with parents who have succeeded in funding, and then actually finding, medical breakthroughs. It can be done. Please help us, his parents and founders of “A Cure for Robert, Inc.”, along with countless friends, board members, and family fill the void that currently exists for new treatments, medicines, and advancements for this very rare disease.
We have to succeed. It’s about our child and thousands of others around the world. There’s simply not another choice.