BRADEN'S STORY

Braden Michael Farrell was born on November 2, 2004 with a head full of 
hair and weighing in at a healthy 8 lbs. 5 oz.  He has a beautiful 
sister, Megan, who is 3 years older than him.  Braden was diagnosed with 
Hurlers syndrome (MPS I) in April 2006.  He had a bone marrow transplant 
at University of Minnesota-Fairview Children's Hospital in Minneapolis 
on September 8, 2006.  His sister, Megan, was the donor.

When Braden was born, we were told that he had to have an ultrasound of 
his spine because there was a patch of hair on his lower back.  We were 
relieved when it came back fine.  We were also told that he had a 
hypospadias and hydroseal (fluid in the testicles).  These would be fixed 
later because hydroseals sometime fixes themselves.  He got RSV when he 
was 5 weeks old and never got over the congestion.  He has suffered with 
many respiratory infections, allergies, and eczema.  Braden also has 
had many ear infections and ended up with tubes twice.  He has had 
pneumonia once and had to have his adenoids removed.  Braden had surgery in 
April of 2006, right before his diagnoses, to repair the hydroseal, 
hypospadias, and a hernia that later appeared.  We also noticed that he was 
slightly developmentally delayed.  Unbeknownst to us, these were 
symptoms of MPS.  Braden's pediatrician made an appointment with a geneticist 
just to rule anything out.  When we received the results of the test, 
we were floored and went through the typical emotions when you receive 
devastating news:  shock, depression, denial, then determination.  We 
found out that there was a chance for him to have a fairly normal life 
and took it.

Affecting 1 in 100,000 children, Hurlers syndrome is a rare genetic 
disorder where the enzyme (alpha-L-iduronidase), which normally breaks 
down the mucopolysaccharides dermatan and heparin sulphate, is missing.  
These mucopolysaccharides build up in all tissues in the body causing 
progressive deterioration and eventual death.

Hurler's children will, without therapy, experience profound mental 
retardation, coarse facial features and excessive hair growth, vision 
problems (due to corneal clouding), and severe heart problems.  Other 
symptoms may include carpel tunnel syndrome, curvature of the spine, 
frequent runny nose, hernias, and hearing loss.  They also rarely live past 
the age of ten years.

To introduce alpha-L-iduronidase into the body, marrow or cord blood 
transplants are being done.  There are also enzyme relacement therapies, 
as well.  Enzyme replacement therapy (ERT) is given through IV infusion 
and would have to be given everyday for the rest of their lives.  The 
drawback to having only ERT, is that it does not go through the 
blood-brain barrier. Therefore, transplant is the other alternative.  
Transplants are performed to provide cells producing enzyme through the growth 
of normal, healthy blood cells.  These blood cells appear to provide 
enzyme to other cells of the brain, preserving neurological function.  
Following successful transplant, patients do not suffer cardiac 
deterioration, and the accumulated mucopolysaccharides in the liver, lungs, and 
marrow slowly disappear.  They may suffer from carpel tunnel syndrome 
and/or back problems later in teens or twenties, in which surgery would 
be needed for repairs.  But, these are minor problems compared to what 
they would suffer without ERT and/or a transplant.

Even though Braden was diagnosed at 17 months old, the doctors said 
that we caught this early, before he suffered any dramatic deterioration.  
Hurlers children usually start deteriorating around the age of 2 years.  
The only issues that he has are the slight hearing problems and the 
small build-up around the mitral valve in his heart.  These are all minor 
issues that can be treated.   He has had 12 weeks of ERT pre-transplant 
and will get 8 weeks of ERT post-transplant.  We believe that this has 
improved his lungs tremendously.  Braden is a very active little boy 
who is progessing wonderfully now. 

We would like to thank all of our family and friends for the tremendous 
support that you have given us.  Please keep us in your prayers!

B.J., Tasha, Megan, and Braden