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This resulted in a prolonged re-coil/ringing phase

PLoS One 9 (3), e93232

Summary The blood-brain barrier is an important structure that separates the brain and spinal cord (the central nervous system, or CNS) from the bloodstream The blood-brain barrier allows only selected molecules to pass, which makes therapy delivery to the brain more difficult Overcoming the blood-brain barrier may involve direct injection into the CNS, adapting a therapy to cross the blood-brain barrier from the blood, or using a delivery vehicle to carry a therapy across More information about therapy options for Sanfilippo can be found on our website

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Some patients developed antibodies that made the medication less effective over time

Staffed by licensed nurses, it runs 24 hours a day, 7 days a week