HURLER'S SYNDROME AWARENESS

HAILEY'S CARING BRIDGE PAGE 

HAILEY'S CARING BRIDGE SITE

Hailey Noel was diagnosed with MPS1/ Hurler Syndrome in Aug. of 2005 at the age of 3 months. Hurler syndrome is a genetic disorder where you're missing an enzyme called Alpha-L-induronidase. This enzyme breaks down sugar molecules in the body called mucopolysaccharides. This disorder is not found till usually about 1 year after life. It's a disorder that slowly happens. Thanks to Duke University Hospital, they were able to save her life, before this disorder took any major effect.

I as Mother with Hailey had spent many days going back and forth to Doctors back home in Virginia Beach to find a solution on what was really going on. It wasn't until December of 2005, I was told 3 options I can either give her a shot every week for Enzyme replacement, or Bone Marrow/Cord Blood Transplant or She can suffer.

I took it up with my Husband we did lots of research on all the options and the best on for the best results and more life saving was a Bone Marrow/Cord Blood Transplant. It was then January 8th 2006 Steven and I and Hailey head off to North Carolina to save our daughters life.

Hailey was admitted to the Hospital right away on January 10th 2006, for the Doctors were concerned about her desating in her oxygen. They watched her very closely. Hailey had 3 major surgical procedures that needed to be done before she could receive her transplant.

On January 20th Hailey under went Anesithia and went into surgery for her Shunt. On January 23rd Hailey went in for her Central line. A line that is inserted in the vessel near the heart to make it easier for IV's and Blood drawing, instead of constantly poking and eventually blowing their veins.

Came early February Hailey went under another surgery to remove her Tonsils and Adenoids. Removing these would give Hailey a bigger airway. For Hurler patients already have a slightly smaller narrower airway.

Thanks to her strength, the great job of the doctors and nurses at Duke Hospital and God's help, she survived ALL this! She is truly a fighter.

Hailey started receiving Chemotherapy February 10th 2006 to prepare her body for her Blood transfusion she was going to receive. On March 1st 2006 Hailey then received her Cord Blood Transfusion from a little new born girl from NYC. That's all we know on the donor. Thanks to the little girl and the care of Dr. Joanne Kurtzburg and her team on 5200 we were able to save Hailey's life. We are thankful for everyday that goes by post transplant. The road after transplant is not easy, but Hailey is fighting everyday to beat the odds against her.

Hailey is now doing great Post transplant and we are now Out of the Hospital, being out Patient and loving everyday and minute of it. We are so close to the end of this long hard road. I give all my thanks and all my Glory to our Lord Jesus Christ and his Angels for with out him, none of this would have been possible for Hailey and I alone.

We will try to keep our friends and family informed thru this journey using this journal. They have been a very important source of support for us. Our greatest inspiration continues to come from Hailey, who never ceases to surprise us with her bravery and courage. She's G-I-JANE!!! She is a Mighty Prayer little lady a warrior walking testimony of Gods power.

Please feel free to sign Hailey's Guest book, we love reading the comments, and Hailey loves hearing from all her family and friends.

HAILEY'S QUILT OF LOVE 

VISIT HERE

MAKAYLA'S CARING BRIDGE PAGE 

MAKAYLA'S SITE

My name is Makayla Alexis Chavers. I was born on July 17, 2006. I left my Mommy and Daddys' arms to be with Jesus on December 17th 2006. I am the baby sister of Post Cord Blood Transplant survivor, Hailey Noel Chavers.

Donations for medical expenses can be sent to:
Hailey & Makayla Chavers Family Trust
C/0 1268 Eagle Way
Virginia Beach, VA 23456

This poem was written by Makayla's Grandmother/MiMi, words from Makayla in Heaven to her family & friends.


PLEASE DON'T CRY FOR ME

Please don't cry for me~
For today, I earned my Angel Wings.
Look! Aren't they pretty?
No more pain or suffering,
Only happiness and singing praises to our King!
I'm having fun playing with all the
little angels that have gone before me,
Running & laughing.
And I'm very happy.
I'll be close by watching over you,
For I came to earth for only a short while
To do the work that God needed me to do.
I was your little girl & your little
KK for just a short while
But please don't remember me being sick
Only remember me as your happy baby
With my big beautiful smile.

I love you~
KK

MAKAYLA'S QUILT OF LOVE 

VISIT HERE

DEFINITION OF HURLER'S SYNDROME 

UNIVERSITY OF MARYLAND MEDICAL CENTER - WEBSITE LINK

Definition:
Hurler syndrome is a rare, inherited disease of metabolism in which a person cannot break down long chains of sugar molecules called glycosaminoglycans (formerly called mucopolysaccharides).

Hurler syndrome belongs to a group of diseases called mucopolysaccharidoses, or MPS.

Causes, incidence, and risk factors:
Persons with Hurler syndrome do not make a substance called lysosomal alpha-L-iduronidase. This substance, called an enzyme , helps break down long chains of sugar molecules called glycosaminoglycans (formerly called mucopolysaccharides). These molecules are found throughout the body, often in mucus and in fluid around the joints.

Without the enzyme, glycosaminoglycans build up and damage organs, including the heart. Symptoms can range from mild to severe.

Hurler syndrome is inherited, which means that your parents must pass the disease on to you. Both parents need to pass down the faulty gene in order for you to develop Hurler syndrome.

Hurler syndrome is a type of mucopolysaccharidosis called MPS I. Hurler syndrome is the most severe type. It is categorized as MPS I H.

The other subtypes of MPS I are:

MPS I H-S (Hurler-Scheie syndrome)
MPS I S (Scheie syndrome

WHAT IS HURLER'S SYNDROME? 

FAIRVIEW - WEBSITE LINK

Affecting one in 100,000 people, Hurler's 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. The disease was first described in 1919 in Germany by Dr. Gertrud Hurler.

Hurler's syndrome babies often develop normally during the first year, but as the storage material (mucopolysaccharides) start to build up, the symptoms begin to appear. Early symptoms include frequent colds and ear infections. Hurler's children will, without therapy, experience profound mental retardation, coarse facial features and excessive hair growth, vision problems (due to clouded cornea), and severe heart problems (as the coronary arteries narrow and the heart valves thicken). Other symptoms may include carpal tunnel syndrome, curvature of the spine, frequent runny nose, hernias, and hearing loss. Most patients die of heart failure between the ages of 5 and 10.

Because Hurler's syndrome is genetic, it is difficult to cure. Current approaches to Hurler's syndrome include genetic counseling for parents who are carriers of the disease, and improvements in early detection of the disease in unborn children (Hurler's can be detected with amniocentesis early in the second trimester). For Hurler's syndrome patients, a variety of treatments have been tried. The goal of treatment is to get the missing enzyme into the body. While the enzyme is now FDA-approved and available, studies have shown this doesn't move into the brain well enough to prevent the neurologic aspects of the disease.

To introduce alpha-L-iduronidase into the body, both gene therapy and blood, marrow or cord blood transplants are being explored. In gene therapy, researchers use a virus to place the gene that produces alpha-L-iduronindase into the patient's cells. 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 neurologic function. However, the enzyme does not appear to penetrate into the brain. Following successful transplant, patients do not suffer cardiac deterioration, and the accumulated mucopolysaccharides in the liver, lungs, and marrow slowly disappear. Vision and hearing generally improve post transplant, as well. Though the mental retardation does not progress, the patient's IQ will typically stabilize. Thus the most successful transplants are those that are performed as soon as possible after diagnosis. Transplants performed after age 2 have disappointment results, because a BMT cannot repair the considerable amount of damage already done.

Because older patients and those with prior lung problems do particularly poorly with transplant, the use of enzyme therapy prior to transplantation may be advantageous. The University of Minnesota has pioneered a study to test the use of enzyme prior to transplant, which is currently enrolling patients.

DUKE PEDIATRIC BLOOD & MARROW TRANSPLANT PROGRAM 

DUKE UNIVERSITY

What is the Duke Pediatric Blood and Marrow Transplant Program?

Since its creation in 1990, the Duke Pediatric Blood and Marrow Transplant (BMT) Program has transplanted over 1200 children suffering from cancer, genetic defects or blood disorders. Today, over half of these children are surviving long-term, cured of their underlying disease by their transplant. With a current waiting list of 30-40 children, and a referral base that is world-wide, the Duke Pediatric BMT Program is the largest children's transplant program in the world, now transplanting some 100 children annually. It is a program of last resort: These children have a life expectancy of less than one year without a transplant.

Pediatric Blood and Marrow Transplantation

The Division of Pediatric Blood and Marrow Transplantation at Duke is an internationally recognized program dedicated to providing outstanding care and support to pediatric patients who can be helped by transplantation therapy. The program is known to be a leader in both established techniques and research initiatives. Under the direction of Joanne Kurtzberg, M.D., the program has performed approximately 1200 autologous and allogeneic transplants in children with cancer or genetic diseases since 1990. Using a variety of approaches the program has pioneered the use of both matched and mismatched umbilical cord blood stem cells in patients who do not have a sibling donor, thus extending this life-saving therapy to many more patients. The cord blood program allows us to identify unrelated donors quickly (generally within a few weeks). We know that sometimes quick identification can be very important for patients with inborn errors of metabolism, bone marrow failure, immunodeficiency syndromes, or advanced leukemias.

The program provides transplant as a treatment option for a variety of acquired and congenital disorders of children and young adults including hematopoietic malignancies and solid tumors, inherited immune deficiency syndromes, bone marrow failure syndromes, and inherited metabolic diseases. Allogeneic and autologous bone marrow, peripheral stem cell and umbilical cord blood transplants have been performed for pediatric patients to treat these diseases.

RAINBOW OF HEROES WALK 

SATURDAY - MAY 2, 2009

RAINBOW OF HEROE'S WALK - WEBSITE LINK

On the first Saturday in May, we walk together as a community. Along the way, as we carry balloons with patients' names on them, people stop, talk together, and enjoy each other. Finally, we release the balloons.

The Rainbow of Heroes Walk commemorates and celebrates all Duke Pediatric Blood and Marrow Transplant (PBMT) patients and their families. Refreshments, music, and activities for everyone are provided.

The Walk is not only the main fundraiser for the Duke PBMT Family Support Program. More than that, it is a reunion for patients, families, nurses, therapists, doctors, volunteers, and supporters who have come to know each other through the months-long transplant process.

2009 RAINBOW OF HEROES WALK - WEBSITE LINK

2009 Walk Plans - Summary about this year's Walk.

when and where: The 2009 Rainbow of Heroes Walk will be Saturday, May 2, 2009, at the Duke Center for Living, Durham, North Carolina, rain or shine. There is no admission charge.

We are supported by our sponsors and our teams' fundraising. Thank you!

We do not have any special arrangement with hotels for this Walk. Participants are responsible for arranging own lodging. We have assembled a list of local hotels for you to make it easier.