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May 11, 2017, 7:30 PM

MPS Awareness Day

 

Dear Church Family,

May 15th is International MPS awareness day.  Mucopolysaccharidoses (MPS) is a genetic lysosomal storage diseases (LSD) caused by a missing or insufficient enzyme that prevents cells from recycling waste, causing materials to be stored in the cells. This leads to widespread damage throughout the body, affecting joints and organ systems. Of the 9 syndrome types, only four have treatments and none has a cure.

Jamie, age 6, was diagnosed with MPS II or Hunter Syndrome in October, 2014. The average life expectancy for boys with Hunter Syndrome is between 10-15 years. There is currently no cure for Hunter Syndrome, however he does receive a weekly IV infusion of an enzyme replacement medication to help flush some of the toxins out of his system.  Jamie currently suffers from hydrocephalus (fluid and pressure on the brain), a mild heart condition, respiratory issues including frequent bronchitis and sleep apnea, and bone and joint pain.  As Jamie gets older, the disease will progress until he no longer eats by mouth, walks, or talks.  Eventually, Hunter Syndrome will take his life.

One of the most promising potential treatments for Hunter Syndrome is gene therapy.  It involves a one-time delivery of a normal copy of the defective gene to allow the body to naturally produce the missing enzyme. Gene therapy has been developed and performed in similar diseases with positive results by doctors at Nationwide Children’s Hospital in Ohio.  These same doctors have developed a similar gene therapy treatment for patients with Hunter Syndrome.  To date, the Hunter Syndrome Foundation and other similar organizations have funded over $500,000 of the research.  An additional $2.5 million dollars is needed to finalize drug production and execute a clinical trial.  

I’m asking our church family to please show support of Jamie and his fight with Hunter Syndrome by wearing Purple on May 15th and by praying for our family and all those affected by MPS. For more information on Hunter Syndrome please visit www.mpssociety.org or Jamie’s Facebook page www.facebook.com/boodersjourney If you wish to make a donation for Gene Therapy either see me or visit https://huntersyndromefoundation.org/donate/what-is-gene-therapy/


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