Satellite Session at the WorldSymposium, 2018

Cognitive impairment in patients with MPS II: From disease burden to cognitive testing, a satellite symposium supported by Shire took place February 2018 at the WORLDSymposium for Lysosomal Storage Diseases. 

Dr. Elsa Shapiro and Dr. Christina Lampe presented on topics including an investigation of challenges in assessing cognition and behavior in patients with MPS II and the burden of cognitive impairment as it relates to daily activity.  During a case study session the audience had the opportunity to vote their responses in an interactive quiz format.



Consensus Papers Posted

In December, 2016 experts from around the globe convened in London for the International Conference for Cognitive Endpoints in MPS. A consensus panel, led by Dr. Hanneke van der Lee, met to address cognitive endpoints in MPS I,  MPS II and MPS III. The resulting literature review and paper now are published and are open access. 

Cognitive and adaptive measurement endpoints for clinical trials in mucopolysaccharidoses types I, II, and III: A review of the literature:

Cognitive endpoints for therapy development for neuronopathic mucopolysaccharidoses: Results of a consensus procedure:

Thank you to the National MPS Society, the MPS Society of the UK and to all our our industry partners for support in making this endevour possible. 

Thank you and Best Wishes to Kate Delaney

While Shapiro & Delaney as a consultancy formed in the past several years, Kate Delaney and Elsa Shapiro have been working together in the rare disease space for more than 20 years. On May 1 we wish Kate well as she takes the next steps in her efforts to help children with rare diseases, leaving the consultancy to make her mark in a new role in industry. 

Everyone who has worked with Kate can attest to her warmth, professionalism, expertise and dedication. We know that Kate will be continue to make a strong impact and further the mission of bringing new and effective treatment to the children who need it. 

Elsa Shapiro will continue to offer expert perspective under the new business name, Shapiro Neuropsychology Consulting. 



EMA Slide Share- Presentation Given at Consensus Conference

Dr. Caroline Auriche, Head of Scientific Advices for the European Medicines Agency (EMA), presented to physicians, clinicians and industry professionals working in Mucopolysaccharidosis (MPS) research at the International MPS Consensus Conference for Cognitive Endpoints.  Slides from this presentation are posted below. 

Slides and Audio: 


Slides (click through as pictures):

Update- A Successful Consensus Conference.

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.



Elsa Shapiro Announced as Keynote Speaker at WORLDSymposium

WORLDSymposium announces Elsa Shapiro, PhD, Professor of Pediatrics and Neurology in the Division of Pediatric Behavioral Neuroscience at the University of Minnesota, as the 2017 Keynote Speaker. Dr. Shapiro's address: Understanding and Measuring Neurodegeneration in Childhood Onset Lysosomal Diseases.

The goal of WORLDSymposium is to provide an interdisciplinary forum to explore and discuss specific areas of interest, research and clinical applicability related to lysosomal diseases. Each year, WORLDSymposium hosts a scientific meeting presenting the latest information from basic science, translational research, and clinical trials for lysosomal diseases. This symposium is designed to help researchers and clinicians to better manage and understand diagnostic options for patients with lysosomal diseases, identify areas requiring additional basic and clinical research, public policy and regulatory attention, and identify the latest findings in the natural history of lysosomal diseases. 

Dr. Shapiro will be speaking on Wednesday, February 15, 2017. 

Learn more:

Shapiro & Delaney form partnership with National MPS Society (U.S.) and UK MPS Society to organize an international consensus conference.

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. 

Contact for more information or with media inquiries. 


FDA Workshop
Held April 16 and 17, this FDA workshop focused on cognitive endpoints in rare inborn errors of metabolism and common diseases. These presentations (video, transcripts, and slides) are now on-line.

UK Mucopolysaccharidosis Society Workshop
The UK Mucopolysaccharidosis Society had an important recent workshop regarding natural history studies and clinical trials in Sanfilippo syndrome (Mucopolysaccharidosis Type III) that included academia, industry, and advocacy groups.  This workshop concluded that common natural history databases and standard protocols were necessary to ensure that patients obtain the best services.