Little Maciek and Great Wizard is the charitable campaign to raise funds for Maciek’s treatment and rehabilitation as well as to support domestic and international medical research centres working on the cure for the Sanfilippo syndrome.
Our goal is to support the following medical research:
Professor Wegrzyn research of genistein aglycone – he proved that the pure genistein slows down the development of the disease in his mice tests. To register the medicine and make it broadly available the clinical tests must be performed.
Research to activate TFEB gene that controls lysosomal enzyme activity – Team Sanfilippo (USA) led by Kathleen Buckley initiated the research aimed to activate the TFEB gene that would result in removing the heperan sulfate improperly stored in the cells of the patient’s body. Currently Team Sanfilippo is pursuing to perform mice tests as soon as possible. The results might be known even within 6 to 12 months. 50 thousand USD is necessary to complete this research.
Gene therapy and the possibility to reverse the Sanfilippo symptoms – the research is performed by Alliance Sanfilippo (Paris, France) led by Karen Aiach. As the clinical tests of gene therapy for MPS IIIA patients are planned, it is necessary to determine if such a treatment may not only stop the development of the disease but also reverse its symptoms which had been occurring since the children birth. The costs of the research amount to 200 thousand EUR.
Our campaign Little Maciek and Great Wizards will include charitable concerts in Poznan and Warsaw and auctions of a number of interesting, “one of a kind” items that we have already collected and that are still to come.
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