2022
Venglustat combined with imiglucerase for neurological disease in adults with Gaucher disease type 3: the LEAP trial
Schiffmann R, Cox TM, Dedieu JF, Gaemers SJM, Hennermann JB, Ida H, Mengel E, Minini P, Mistry P, Musholt PB, Scott D, Sharma J, Peterschmitt MJ. Venglustat combined with imiglucerase for neurological disease in adults with Gaucher disease type 3: the LEAP trial. Brain 2022, 146: 461-474. PMID: 36256599, PMCID: PMC9924909, DOI: 10.1093/brain/awac379.Peer-Reviewed Original ResearchConceptsGaucher disease type 3Years of treatmentEnzyme replacement therapyWeek 26Biomarker reductionLEAP trialWeek 52Patients 9Neurological manifestationsReplacement therapyPlasma concentrationsType 3Day 1Brain volumeAcid β-glucosidase activityImiglucerase enzyme replacement therapyDiverse neurological manifestationsSystemic disease parametersSerious adverse eventsInfiltrative lung diseaseWhole brain volumeRegional brain activityExploratory endpointsPrimary endpointSecondary endpoints
1989
INHIBITION OF PLATELET AGGREGATION BY ABNORMAL HIGH DENSITY LIPOPROTEIN PARTICLES IN PLASMA FROM PATIENTS WITH HEPATIC CIRRHOSIS
Desai K, Bagget C, Bellamy M, Mistry P, Burroughs A, Owen J. INHIBITION OF PLATELET AGGREGATION BY ABNORMAL HIGH DENSITY LIPOPROTEIN PARTICLES IN PLASMA FROM PATIENTS WITH HEPATIC CIRRHOSIS. The Lancet 1989, 333: 693-695. PMID: 2564508, DOI: 10.1016/s0140-6736(89)92207-1.Peer-Reviewed Original Research