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4:39
YouTube
Osmosis from Elsevier
Sanfilippo syndrome (Year of the Zebra)
What is Sanfilippo syndrome? Sanfilippo syndrome, also known as mucopolysaccharidosis type III, or MPS III for short, is a rare genetic metabolic disorder which ...
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Sanfilippo Syndrome — also known as Mucopolysaccharidosis type III or MPS III — is a terminal, neurodegenerative rare disease. It causes children to lose all the skills they’ve gained, suffer seizures and movement disorders, experience pain and suffering, and then die, often before the second decade of life. Because of its neurodegenerative nature and multi-system impact, Sanfilippo Syndrome is often called “childhood Alzheimer’s” or “childhood dementia.” Currently there is no FDA-approved treat
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