Publications by authors named "Lara Longpre"
Article Synopsis
- Progressive familial intrahepatic cholestasis (PFIC) is an autosomal recessive disorder mainly caused by BSEP deficiency, leading to issues like bile formation disruption and severe itching (pruritus).!* -
- The MARCH-PFIC study was a phase 3, double-blind, placebo-controlled trial involving participants aged 1-17 from 29 centers across 16 countries, focusing on the effects of the drug maralixibat on different types of PFIC.!* -
- The study aimed to measure changes in pruritus severity and serum bile acid levels over 26 weeks, specifically looking at outcomes in cohorts with varying forms of PFIC, comparing those treated with maralixibat
Background: Intraputamenal glial cell line-derived neurotrophic factor (GDNF), administered every 4 weeks to patients with moderately advanced Parkinson's disease, did not show significant clinical improvements against placebo at 40 weeks, although it significantly increased [18F]DOPA uptake throughout the entire putamen.
Objective: This open-label extension study explored the effects of continued (prior GDNF patients) or new (prior placebo patients) exposure to GDNF for another 40 weeks.
Methods: Using the infusion protocol of the parent study, all patients received GDNF without disclosing prior treatment allocations (GDNF or placebo).
We investigated the effects of glial cell line-derived neurotrophic factor (GDNF) in Parkinson's disease, using intermittent intraputamenal convection-enhanced delivery via a skull-mounted transcutaneous port as a novel administration paradigm to potentially afford putamen-wide therapeutic delivery. This was a single-centre, randomized, double-blind, placebo-controlled trial. Patients were 35-75 years old, had motor symptoms for 5 or more years, and presented with moderate disease severity in the OFF state [Hoehn and Yahr stage 2-3 and Unified Parkinson's Disease Rating Scale motor score (part III) (UPDRS-III) between 25 and 45] and motor fluctuations.
View Article and Find Full Text PDFOncolytic viruses and active immunotherapeutics have complementary mechanisms of action (MOA) that are both self amplifying in tumors, yet the impact of dose on subject outcome is unclear. JX-594 (Pexa-Vec) is an oncolytic and immunotherapeutic vaccinia virus. To determine the optimal JX-594 dose in subjects with advanced hepatocellular carcinoma (HCC), we conducted a randomized phase 2 dose-finding trial (n=30).
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