(Bentham Science Publishers) The rare, chronic, autosomal-recessive lysosomal storage disease Niemann-Pick disease type C1 (NPC1) is characterized by progressively debilitating and ultimately fatal neurological manifestations. There is an urgent need for disease-modifying therapies that address NPC1 neurological pathophysiology; and passage through the blood-brain barrier represents an important consideration for novel NPC1 drugs.
from EurekAlert! - Social and Behavioral Science http://ift.tt/2nZv1JK
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