Parents your children could help the research for a cure / treatment for Sanfilippo syndrome, it is frequently difficult to obtain such tissues for some genetic diseases but scientists can go to tissue and cell banks. Here they can select the samples they need for a specific disease from patients.
Sanfilippo syndrome research might be improved if we could get more samples of such tissues with different mutations into biobanks.
There are several biobanks. One example is the Coriell Institute for Medical Research which is an independent, nonprofit research organization that houses the world’s largest collection of human cell lines. They make them available to researchers around the world for a nominal cost and they can do this because they are supported by grants from the National Institutes of Health and several private foundations with specific support by the National Institute of General Medical Sciences.
You can find out much more about Coriell at their website at http://ccr.coriell.org. Also they have specific information for patients https://catalog.coriell.org/1/NIGMS/About/Information-for-Patients. All interested families will need to sign the Coriell consent forms in order to have their samples in the repository. Informed Consent form Assent form.
A very difficult decision for a rare disease parent is to donate a sample of brain tissue and other tissues after their passing for future medical research. The NIH have funded a network of Brain and Tissue Banks referred to as the NIH NeuroBioBank. Each of the banks collects and distributes tissue from donors. More information about the operation of the NIH NeuroBioBank is provided on the Tissue Requests page. The UMB Brain and Tissue Bank is located at the University of Maryland School of Medicine, Department of Pediatrics in Baltimore, Maryland (http://medschool.umaryland.edu/BTBank/). The mission of the UMB BTB is to advance the research of developmental, neurological and movement disorders.
There are certainly many other resources available but these should provide an introduction and raise awareness that we can provide our researcher community with the cells and tissues they need to do their work.