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Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy

LYSOGENE · 2013
ClinicalTrials.gov · Datasets · License: Public Domain · 2013
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genetherapygeneticdiseasesmetabolicdiseasesneurodegenerativediseases
mucopolysaccharidosis type iii a, sanfilippo disease type a, neurodegenerative diseases, nervous systems diseases, genetic diseases, inborn, metabolic diseases, gene therapy, adeno associated virus (aav), interventional, phase1, phase2
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