August 21, 2020

Rare Disease: Spinal Muscular Atrophy

Date and time

August 20, 2020 ~ August 20, 2020

10:30am – 12:30pm EDT | 4:30pm – 6:30pm CET

August is the month each year when special efforts are made to raise awareness of patients suffering from Spinal Muscular Atrophy (SMA), a crippling, degenerative neuromuscular disease. In its most severe form, SMA is the leading genetic cause of infant death. WuXi AppTec welcomes the opportunity to contribute to SMA Awareness Month this year through our next webinar, “Progress in Halting Spinal Muscular Atrophy”, on Aug. 20, 2020.

This is the third webinar in WuXi AppTec’s “Collaborations That Transform” series on rare diseases, where we convene physicians, drug developers, patients, and advocates to critically examine the current state of therapies and the promise of the next generation of treatment approaches and modalities

We would like to thank BioCentury, Children’s Tumor Foundation, CLSA, Informa, New England Hemophilia Association and UK Bioindustry Association for their great support of the “Collaborations That Transform” webinar series.

Agenda

10:30 AM - 10:35 AM Welcome
Hui Cai, VP and Head of Content, WuXi AppTec, Program Chair
10:35 AM - 10:55 AM Session I: Perspective on Global Development and Access to Medicines in Rare Diseases
A conversation with Paulo Fontoura, Global Head and SVP, Neuroscience and Rare Diseases Clinical Development, Roche; moderated by David Chang, CEO, WuXi Advanced Therapies, WuXi AppTec
10:55 AM - 11:35 AM Session II: Patients at the Center
Kristin Schneeman, Director, FasterCures, a center of the Milken Institute
Mencía de Lemus, President of SMA Europe; President of FundAME, National SMA patient organisation, Spain
Laurent Servais, Professor of Paediatric Neuromuscular Diseases, Neuromuscular Reference Center Disease, Department of Paediatrics, Liège, Belgium; MDUK Neuromuscular Center, Department of Paediatrics, University of Oxford, UK
Moderated by Lauren Martz, Senior Editor, Head of Translation & Clinical Development, BioCentury
11:35 AM - 12:25 PM Session III: Translating Fundamentals into Meaningful Therapeutics
Karen Chen, CEO, Spinal Muscular Atrophy Foundation
Stanley Crooke, Chairman of the Board and CEO, Ionis Pharmaceuticals; Founder, CEO and Chairman of the Board, n-Lorem Foundation
Thomas Crawford, Professor of Neurology and Pediatrics, Johns Hopkins Hospital, Johns Hopkins Children’s Center, Kennedy Krieger Institute
Oscar Henry Mayer, Professor of Clinical Pediatrics, The Perelman School of Medicine at the University of Pennsylvania, Division of Pulmonology, Children’s Hospital of Philadelphia
Moderated by Richard Soll, Head of WuXi AppTec’s Boston office; Senior Advisor of Strategic Initiatives
12:25 PM - 12:30 PM Closing

Speakers

Hui Cai
VP and Head of Content Division, WuXi AppTec
Karen Chen
CEO, Spinal Muscular Atrophy Foundation
Thomas Crawford
Professor of Neurology and Pediatrics, Johns Hopkins Hospital, Johns Hopkins Children’s Center, Kennedy Krieger Institute
Stanley Crooke
Chairman of the Board and CEO, Ionis Pharmaceuticals; Founder, CEO and Chairman of the Board, n-Lorem Foundation
Paulo Fontoura
Global Head and SVP, Neuroscience and Rare Diseases Clinical Development, Roche
Mencía de Lemus
President of SMA Europe; President of FundAME, National SMA patient organisation, Spain
Oscar Henry Mayer
Professor of Clinical Pediatrics, The Perelman School of Medicine at the University of Pennsylvania, Division of Pulmonology, Children’s Hospital of Philadelphia
Kristin Schneeman
Director, FasterCures, a center of the Milken Institute
Laurent Servais
Professor of Pediatric Neuromuscular Disease, MDUK Oxford Neuromuscular Centre; Head of Specialized Translational Research Oxford Neuromuscular Group: Invited Professor of Child Neurology, Liège University
Lauren Martz
Senior Editor, Head of Translation & Clinical Development, BioCentury
David Chang
CEO, WuXi Advanced Therapies, WuXi AppTec
Richard Soll
Head of WuXi AppTec’s Boston office; Senior Advisor of Strategic Initiatives