This Thursday is International MPS Day. Wear purple in support of Coleson and all other MPS families.
Also, today was Coleson's last dressing change. Hooray! Next Tuesday (5/20) he goes in for his line removal and port placement. He will finally be able to take a real bath and go for a real swim!
Here is a short You Tube video narrated by Alec Baldwin on lysosmal disorders.
Here is some info on MPS as provided by another MPS mother.Thanks
for MPS I (Hurler syndrome): Successful HSCT (Hematopoietic Stem Cell
Transplantation) has been performed for children with Hurler syndrome since
1980. The immediate benefits include correction of the enzyme deficiency and clearance of glycosaminoglycans (GAGs). Long-term benefits include the
possibility of long-term survival by protecting the heart, lungs, and brain
from the effects of progression of the MPS disorder. Other organs and tissues
can also show benefits from the HSCT; these include the eyes and ears, liver,
spleen, joints, airway, etc. However, it should be noted that many children are
still requiring a variety of orthopedic surgeries despite a successful transplant.
While the term “cure” should not be used, HSCT has the longest track record of
any effective therapy for Hurler syndrome, including the ability to preserve
cognitive function and development in the normal range.
Brought to you by Be The Match: WWW.BeTheMatch.OrgA bone marrow or cord blood transplant is the only treatment
that can stop the effects of Hurler syndrome at this time. A bone marrow or
cord blood transplant begins with chemotherapy, with or without radiation, to
destroy the diseased cells and marrow. The transplant replaces diseased
blood-forming cells with healthy ones.
The type of transplant
used for Hurler syndrome is an allogeneic transplant. This type of transplant
uses healthy blood-forming cells from a family member, unrelated donor, or
umbilical cord blood unit.
For an allogeneic transplant, a patient gets chemotherapy, with
or without radiation, prior to transplant to prepare his or her body for the
treatment. Then the replacement cells are infused into the patient’s blood
stream. From there, the cells find their way into the bone marrow, where they
start making healthy red blood cells, white blood cells and platelets. These
new cells have the enzyme needed to break down GAG and stop further damage to
It is because of the
shortcomings of the current treatments (and the lack of treatments entirely)
that organizations such as The National MPS Society, Hunter Syndrome
Foundation, Team Sanfillipo, Hunter Syndrome Research Coalition, Ryan Dant
Foundation, and numerous other parent based organizations exist. If it were not
for the willingness of families to not only say “Something has to change” but
also to stand up and do something about it, doctors like Emil Kakkis would have
lost funding for research before they were able to bring a treatment to reality
for us. These family organizations work not only at funding promising new
research, but also at advocating inside of the FDA and congress to educate on
MPS, and negotiate the best ways to bring safe and effective treatments through
the system as quickly as possible so that families can gain access before it is
too late to help their child.
This year International MPS Awareness Day falls on a Thursday
(May 15th) so many families will be celebrating with fundraising events today.
The success these events translates to saving the lives. It translates to hope.
So make sure you ask the MPS Family you love how you can help give them hope
“What we needed was one day. Whatever money you could raise in
one day, somewhere somebody could use it.” -Mark Dant MPS I HS Dad