Our History

ADAM & LEA – OUR HISTORY


WHO ARE WE

This story is about us:  Adam and Lea, siblings connected in struggle for life against MPS 3 Sanfilippo.

My name is Adam, but you may call me Adas. And ‘this small thing’ is Lea – my sister. It is no so long ago that we entered the world and we’ve already had the first serious flop… Sanfilippo. What can we say – each of us had a 25% of having this defect. And guess what – both of us have it. Well, what can we do… our parents made their research and it turned out that there is hope for us! But this topic is tackled in other sections of this website. Here you will find a little bit of information about us. Although we have this crap disorder, we are very cool and adorable kids.

HISTORY

PROBLEM

We have a genetic defect (you would never know from looking our photos) and we need to do something about it. We are lacking one enzyme (seems no problem, but still) that breaks down some complex sugars.

PLAN

There are three options (starting with the best one):

  • To replace the gene and be cured for good (gene therapy)
  • Give us the missing enzyme (enzyme replacement therapy)
  • Slowing down production of these sugars (Genistein)

SUCCESS

  • Collect the money
  • Organise patients
  • Support the doctors
  • And defeat this damn MPS3

ADAM AND LEA HELP US TO MAKE A CHANGE