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.

Dr. Kendra Bjoraker, Dr. Kelly King, and Dr. Julie Eisengart present on Patient/Parent Reported Outcomes.

Dr. Brian Bigger and Kate Delaney moderate a lively question and answer session.

The Consensus Panel, facilitated by Dr. Hanneke van der Lee.

Dr. Brian Bigger co-presented workshop sessions.

Dr. Elsa Shapiro presents. Dr. Patroula Smpokou, FDA; Dr. Hanneke van der Lee, Academic Medical Center, Amsterdam, The Netherlands ; Dr. Darren Janzen, Oregon Health Science University, United States; Kate Delaney, Shapiro & Delaney LLC; and Dr. Caroline Auriche, EMA on stage.

The consensus conference was organized by Kate Delaney and Dr. Elsa Shapiro, to either side of Dr. Hanneke van der Lee, Pediatric Clinical Research Office, Academic Medical Center, Amsterdam, The Netherlands, who oversaw the consensus process.

Dr. Chester Whitley presents on MPS disorders: Genotype- Phenotype Correspondence.

Dr. Maureen Cleary presenting.

Dr. Hernan Amartino presenting.

Dr. Igor Nestrasil presents on the relationship of neuroimaging to cognition.