dysarthria/癲癇性発作

Niemann-Pick disease type C (NPC) is a lipid storage disease that can present in infants, children, or adults. Neonates can present with ascites and severe liver disease from infiltration of the liver and/or respiratory failure from infiltration of the lungs. Other infants, without liver or

Neurologic disorders that affect the liver transplantation candidates when they are on the waitlist not only significantly affect preoperative morbidity and even mortality but also present important predictive factors for post-op neurologic manifestations. Attentive neurological evaluation before

This is a Phase 1, multi-center, open-label study designed to evaluate the safety and pharmacokinetics of EXPAREL when administered via local wound infiltration to subjects undergoing open cervical or thoracic spinal fusion or reconstructive surgery. Fifteen subjects are planned for enrollment.

This is a Phase 4, multicenter, randomized, double-blind, controlled study in approximately 220 adult subjects undergoing primary, 1-2 level, open lumbar spinal fusion surgery under general anesthesia. Subjects will be screened within 30 days prior to study drug administration and at least one day

Niemann-Pick Disease, type C (NPC) is an autosomal recessive lysosomal storage disease with progressive neurodegeneration. It is characterized by intracellular accumulation of cholesterol and glycosphingolipids. The age of onset is variable with cases manifesting from infancy to adulthood.

Niemann-Pick Tyoe C disease (NPC) is an autosomal recessive, lysosomal storage disorder characterized by accumulation of cholesterol and gangliosides. NPC is a rare (estimated prevalence of 1:120,000-150,000) neurodegenerative disorder with a wide clinical spectrum and a variable age of onset.