preview

Sanfilippo Case Studies

Satisfactory Essays

Please help find a CURE Spencer is 2 years old and was just diagnosed with a rare, progressive, terminal, genetic disease, Sanfilippo – MPS III. Children with Sanfilippo do not show signs of the disorder at birth. With the progression of the disease the children, degenerate, loosing the ability to speak, walk, eat and eventually loose their lives. Families of Sanfilippo children have come together raising money for research and to start clinical trials. Without the parents doing fundraisers there wouldn't be any clinical trials because the disease is so rare the drug companies haven't wanted to invest any money yet. There is a gene therapy clinical trial going on now that is showing promising hope. Three children were treated

Get Access