Shapiro & Delaney, together with the National MPS Society and the UK MPS Society, are organizing an international consensus conference on neurocognitive and related endpoints in clinical trials for the mucopolysaccharidoses.
Across the spectrum of MPS disorders, differing endpoints in natural history studies and clinical trials lead to lack of comparability of results, which handicaps the identification of effective treatments. With the development of new treatments, it is crucial to develop a guide for future studies.
To be held Dec 2 and 3 in the UK, with more than 70 invited international experts attending, this consensus conference will address cognitive endpoints in MPS I, II and III. Results from the conference Delphi proceedings, as well as a Literature review and industry survey will be compiled and published. In addition to Consensus Panel proceedings, the conference will serve as a key touch-point for industry representatives and invited attendees with Kate Delaney leading panel discussions in pertinent topics such as: obstacles to accurate neurocognitive assessments, early developmental assessments of infants and regulatory considerations when using neurocognitive endpoints in clinical trials.
Conference Steering Committee:
Elsa Shapiro, PhD, Shapiro & Delaney, LLC, & University of Minnesota, USA; Mark Dant, US National MPS Society; Kathleen Delaney, Shapiro & Delaney LLC; Christine Lavery, UK Society for Mucopolysaccharide Diseases; Brian Bigger, PhD, University of Manchester, UK; Patricia Dickson, MD, Harbor-UCLA Pediatrics, Los Angeles, CA, USA; Maria L Escolar, MD. MS. University of Pittsburgh, PA, USA; Roberto Giugliani,MD, PhD, Federal University of Rio Grande do Sul, Porto Alegre, Brazil; Paul Harmatz, MD, UCSF Benioff Children's Hospital Oakland, CA, USA; Simon Jones, M.D., St. Mary’s Hospital, Central Manchester University Hospitals, University of Manchester, UK; Shauna Kearney, Ph.D., Birmingham Children's Hospital, UK; Joseph Muenzer, MD, PhD, University of North Carolina at Chapel Hill, USA; Igor Nestrasil, MD, PhD, University of Minnesota, USA; Maurizio Scarpa, MD, Horst Schmidt Clinic, Wiesbaden, Germany; Frits Wijburg, MD, PHD Academic Medical Center, U of Amsterdam, Netherlands; Hernan Amartino, MD. Hospital Universitario Austral, Buenos aires, Argentina; Simon Jones, MD, Central Manchester University Hospitals, University of Manchester, UK; Lorne Clark, MD, University of British Columbia, Canada; Stewart Rust, PHD, Royal Manchester Children's Hospital, UK
This conference is possible through the generous support of eighteen pharmaceutical industry sponsors.
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