A clinical trial for Sanfilippo Syndrome Type A is currently enrolling patients.

The Lyrics Study, HGT-SAN-093, is evaluating the safety and efficacy of intrathecal delivery of an enzyme replacement therapy on cognitive function in pediatric patients with Early Stage MPS IIIA Disease (Sanfilippo Syndrome Type A.) There are currently no effective disease therapies currently approved for this devastating and disabling disease.

Each patient must meet the following criteria to be enrolled in this study.

1. Documented MPS IIIA diagnosis.

2. Between 12 and 48 months of age at the time of enrollment.

3. The patient has a DQ score equal to less than 60%.

IF you have interested in the study and would like to learn more you can contact Dr. David Alexanderian directly at Dalexanderian@shire.com.



2 thoughts on “Clinical Research Study on Sanfilippo Syndrome Type A (MPS III)”

  1. jens says:

    Life with incurable disease MPS 3
    Mucopolysaccharidosis, homepage of the family, unsergoldkind.npage.de
    ……………………………………………………………………………………………………..Die Confrontation with the diagnosis was probably devastating to parents. The list of problems may give the impression that life is very overshadowed with Sanfilippo child problems. But it is quite possible to help the affected children to live a happy and fulfilling life. Parents talk about their positive feelings:
    Many Sanfilippo kids like music, especially if she is beautiful rhythmically. and they like to watch TV advertising, Spongebob and animal documentaries.
    Sometimes I wonder what they think probably if you look around your eyes look so knowingly and with its rays may emit a kind of consolation. I personally believe that we often simply do not understand the language of these children. We can ill detach ourselves from our traditional ideas about how our child should be, and thus are a real understanding himself in the way.
    I believe that our sick children experience a lot more than we give them credit for. I even think it is not impossible that in certain stages of the disease, a part of the aggression comes from the fact that the child from the environment (either by the family or by strangers) is not taken seriously or is repelled. Would be a healthy child in such situations not behave so?
    Many parents of MPS children have to deal with a really hard road! they often can not cope without outside help

  2. Wth?? I think you need some help. Evidently your having some coping issues. Speak for yourself. Not all mps 3 children are the same…just like not every healthy child is the same . every chid is different, unique, special. God gives the extra special childrne, whom need extra special help ….to extra special parents…..you should think through what you plan on writing before you act on it next time. Your post was extremely aggravating and even more irrigating.

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