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:

http://www.mgmjournal.com/article/S1096-7192(17)30209-3/fulltext

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

http://www.mgmjournal.com/article/S1096-7192(17)30211-1/fulltext

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. 

Accelerating the Conversation: Rare Disease, The Patient Experience and What's Next

Elsa Shapiro and Cara O’Neill, Co-founder and Vice President of Cure Sanfilippo Foundation discuss the future of research, the need for collaboration and more.

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):

Video Share- Presentation from MPS Consensus Conference Workshop

Dr. Igor Nestrasil, University of Minnesota, presents Part 1 Cognition and Neurological Outcomes of the workshop session: Neurological, Neuroimaging, and Biochemical Markers and their Relationship to Cognition. Includes audience comments and discussion. 

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.

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.