Sanfilippo Syndrome Registry Project and Natural History Studies: An Example of Patients, Parents and Researchers Collaborating for a Cure Jill Wood1,2 , Stuart Siedman3, Jennifer Siedman3, Paul Levy4, Kyle Brown5, Kim M

Source URL: www.rare-diseases.eu

• Rare diseases
• Syndromes
• Sanfilippo syndrome
• Mucopolysaccharidosis
• Lysosomal storage disease
• Clinical trial
• Dementia
• Gene therapy
• Sylvester Sanfilippo
• Health
• Medicine
• Biology