In partnership with the National MPS Society (U.S.) and UK MPS Society, Shapiro & Delaney organized a successful consensus conference to discuss neurocognitive and related endpoints in clinical trials for the mucopolysaccharidoses.
Held in London in the beginning December, this conference drew 80 invited international experts as well as representatives from 18 pharmaceutical companies. Parents and patient advocates were also represented.
Both FDA and EMA representatives attended and presented.
The conference format consisted of large group presentation of current literature by Dr. Darren Janzen and discussion by experts and audience, followed by a second day of Delphi consensus panel deliberations that were open for observation.
Workshop sessions were also held on the second day. Overseen by Kate Delaney and Dr. Brian Bigger, presented pertinent topics and brought experts from around the world together with industry for open and productive discussion. Sessions included:
- MPS Disorders: Clinical Manifestations in MPS I, II and III- Dr. Maureen Cleary
- MPS disorders: Genotype-Phenotype Correspondence- Dr. Chester Whitley
- New Treatment Options and Ongoing Clinical Trials- Dr. Brian Bigger
- Patient/Parent Reported Outcomes- Dr. Julie Eisengart, Dr. Kendra Bjoraker, Dr. Kelly King
- Newborn Screening and Early Identification; Challenges for Treatment and Assessment – Dr. Paul Orchard, Kate Delaney
- The Basics of Reliable Cognitive Assessment Kathleen Delaney, -J.P. Marchal
- Neurological, Neuroimaging and Biochemical Markers and their Relationship to Cognition- Dr. Igor Nestrasil, Dr. Hernan Amartino, Dr. Patricia Dickson
Workshop presentation videos will be released for viewing beginning in mid-January.
Consensus proceedings, overseen by Dr. Hanneke van der Lee at the University of Rotterdam, resulted in a list of recommendations and assessment guidelines. Papers describing the results will be submitted to journals in the first quarter of 2017 and will be touched on in Dr. Elsa Shapiro's keynote addess at the WORLDSymposia in February 2017.