Assessment of Target Engagement in a First‐in‐Human Trial with Sinbaglustat, an Iminosugar to Treat Lysosomal Storage Disorders by Martine Gehin, Meggane Melchior, Richard W.D. Welford, Patricia N. Sidharta, Jasper Dingemanse

Get full text

Novel high-throughput myofibroblast assays identify agonists with therapeutic potential in pulmonary fibrosis that act via EP2 and EP4 receptors. by Patrick Sieber, Anny Schäfer, Raphael Lieberherr, François Le Goff, Manuel Stritt, Richard W D Welford, John Gatfield, Oliver Peter, Oliver Nayler, Urs Lüthi

Get full text

Plasma lysosphingomyelin demonstrates great potential as a diagnostic biomarker for Niemann-Pick disease type C in a retrospective study. by Richard W D Welford, Marco Garzotti, Charles Marques Lourenço, Eugen Mengel, Thorsten Marquardt, Janine Reunert, Yasmina Amraoui, Stefan A Kolb, Olivier Morand, Peter Groenen

Get full text

Quantification of increased biologically active CXCL12α plasma concentrations after ACKR3 antagonist treatment in humans by Peter Blattmann, Hervé Farine, Brian Dan, Nicole Stiffler, Thomas Lefebvre, Christine Huynh, Patricia N. Sidharta, Jasper Dingemanse, Richard W. D. Welford, Marcel Keller, Daniel S. Strasser

Get full text

Plasma neurofilament light, glial fibrillary acidic protein and lysosphingolipid biomarkers for pharmacodynamics and disease monitoring of GM2 and GM1 gangliosidoses patients by Richard W.D. Welford, Herve Farine, Michel Steiner, Marco Garzotti, Kostantin Dobrenis, Claudia Sievers, Daniel S. Strasser, Yasmina Amraoui, Peter M.A. Groenen, Roberto Giugliani, Eugen Mengel

Get full text