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Aug
14

A needle in a haystack

A busy day before going to the ACS. We have put a couple of similar posts (here and here) out in an attempt to try to identify some Sanfilippo type D (MPS IIID) patients or at the very least to get their families to register in the registry .

This is like finding a needle in a haystack. Looking through the literature there are so few case reports and certainly nothing recently, which calls out for an update. If you are reading this and know of any, please point them our way and we can let them know there is work ongoing to develop an enzyme replacement.

I would certainly welcome any ideas of how we could find these families.

 

1 comment

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  1. Greta says:

    Have you contacted CureSFF.org?
    They have 823 followers on twitter….
    @SavingEliza

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