Speakers

Orphan Drugs & Rare Diseases 2020

Bob Stevens

Group CEO, MPS Society

Dr. David A Walker

B Med Sci FRCP FRCPCH, Professor of Paediatric Oncology, Children’s Brain Tumour Research Centre

Didier Caizergues

Head, Regulatory Affairs Department, GENETHON

Edny Inui

Director, Business Development, Caribou Biosciences

Eugean Jiwanmall

Sr Research Analyst, Independence Blue Cross

Dr. Fatima Sulaiman

Head of Research and Services, Scleroderma & Raynaud's UK

Joanne M. Hackett

Chief Commercial Officer, Genomics England

Dr. Janet Allen

Director of Strategic Innovation, Cystic Fibrosis Trust

Thomas Kuhler

Head Regulatory Science & Policy, EU/AMEE, Sanofi

David Gillen

Vice President, International Medical Affairs, Vertex

Sheela Upadhyaya

Associate Director - Highly Specialised Technologies, NICE

Sonali Chopra

Director, Alliance and Advocacy Relations, Genentech, Inc.

Gulin Donmez

General Manager, Turkey, Amryt Pharma

Femida Gwadry-Sridhar

Chief Executive Officer and Founder, Pulse Infoframe

Leslie Galloway

Expert Adviser, NICE, Chairman, Ethical Medicines Industry Group

Dr. Nicolas Sireau

CEO and Chair of Trustees, AKU Society

Dr. Rohit Batta

Chief Medical Officer, Vicore Pharma Holding AB

Dr. Linda Popplewell

Department of Biological Sciences, Royal Holloway University of London

Martina Garau

Director, OHE

Karen Facey

Sr. Research Fellow, Impact HTA - Co-Lead Investigator, WP10

Sylvie Gabriel

SVP, Global Market Access & Pricing, Ipsen

Robert Johnstone

Patient Advocate - International Foundation for Integrated Care (IFIC), European Forum for Good Clinical Practice (EFGCP) & Health Quality Improvement Partnership (HQIP)

Karen Matthews

Business Development Manager NIHR

Paolo Morgese

EU Director Market Access, Alliance for Regenerative Medicines

Emily Crossley

Co-founder and joint CEO, Duchenne UK

Rafał Świerzewski

President of Board, Fundation for Patients with Rare Diseases COCARDA

Rosa Oliveira

Senior Director, Head Commercial Neurology, EMEA, Lupin Neurosciences

Edmund Lim

Founder and President, WeCareJourney.org

Jim Green

Chairman of Board of Trustees of INPDR (International Niemann-Pick Disease Registry)

Kerry Leeson-Beevers

National Development Manager & Project Lead, Alström Syndrome

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Bob Stevens

Group CEO, MPS Society

Bob Stevens is the Group CEO of MPS Society & MPS Commercial, he is also Co-Chair of the International MPS Network and Vice-Chair of the LSD Collaborative. Bob’s previous roles include being the Managing Director of a charity supporting people of all ages with learning disabilities and complex needs and indeed he was a Trustee for the MPS Society for over 10 years.

Prior to being involved within the not for profit sector Bob had a successful career in the commercial sector within construction. Bob spent over 10 years operating at Board level alongside his private property development portfolio.


Bob has two sons with MPSII (Hunter’s Syndrome) which is one of the rare genetic diseases that is supported by the MPS Society, he says of his role that it is not a job but ‘a way of life’.

Dr David A Walker

B Med Sci FRCP FRCPCH, Professor of Paediatric Oncology, Children’s Brain Tumour Research Centre

David was born in Scotland and qualified in Medicine from University of Nottingham in 1977 subsequently training in adult and paediatric medicine in UK (Cambridge, St Mary’s Paddington, Leicester, Sheffield, Great Ormond Street Hospital and Royal Children’s Hospital, Melbourne Australia), being appointed as a Senior Lecturer in Paediatric Haematology and Oncology in Nottingham in 1990 and appointed as Professor in 2005.

He led the children’s brain tumour trials’ committees in UK (CCLG 92-97) and Europe (SIOPe 97-2001) and co-chaired the International Consortium of Childhood Low Grade Glioma 1997 – 2014. He co-chaired the Royal College of Paediatrics and Child Health (RCPCH), Society of British Neurological Surgeons (SBNS)and Royal College of Nursing (RCN) working party to establish a network of children’s brain tumour treatment centres across the UK reporting in 1997. He helped to develop the Children’s Brain Tumour Research Centre at the University of Nottingham http://www.cbtrc.org/. It celebrated its 20th anniversary in 2017. He helped initiate the Teenage Cancer Trust (TCT) adolescent units in East Midlands Children and Young People’s Cancer Service (EMCYPICS) and was a TCT Visiting Professor to Royal Children’s Hospital in Melbourne, Australia in 2007.

Didier Caizergues

Head, Regulatory Affairs Department, GENETHON

A Doctor in Pharmacy, who holds a degree in Health law. He enjoyed a long career in the field of Regulatory Affairs.


He began his career as a consultant with International Drug Development (IDD), but soon joined the company SYNTHELABO in its Research and Development center where he worked for nine years. In this office Dr Didier CAIZERGUES oversaw the registration of new molecules that had evolved from cardiovascular, urological and anti‐depressive research.

He later headed up the international registration department for the Pierre Fabre group and for the next six years he practices to the fields of anti‐cancer drugs, anti‐depressants and orphan diseases. In 2001 he joined the GENETHON as Director of International Regulatory Affairs where he set up a regulatory affairs department specialized in gene and cell therapies in the fields of rare diseases. He obtained several clinical trials authorization in European countries and US for gene therapy products with lentivirus and AAV in the fields of muscular dystrophies and paediatric immunodeficiencies. He also manages scientific advice with the European Medicines Agency. He is also in charge of the assessment of new molecules or molecules already marketed still in the field of rare diseases in different working groups in European projects (Treat‐NMD).

Edny Inui

Director, Business Development, Caribou Biosciences

Edny Inui, PhD, MBA is the Director of Business Development at Caribou Biosciences, a biotech company founded out of UC Berkeley by CRISPR pioneers Jennifer Doudna, Martin Jinek, and Rachel Haurwitz. Edny leads the out-licensing of Caribou’s CRISPR genome engineering technologies in addition to strategic in-licensing and partnering of new technologies in support of the Caribou portfolio of allogeneic CAR-T cell therapies. She has spent over a decade in the biotechnology industry with prior roles as Senior Manager, Business Development and Strategy at Acadia Pharmaceuticals and as a Senior Biotechnology Analyst for Biomedtracker, Informa Pharma Intelligence. She holds a BS in Biology from the Massachusetts Institute of Technology, a PhD in Neuroscience from the University of Michigan, and an MBA from the University of Chicago Booth School of Business.

Eugean Jiwanmall

Sr Research Analyst, Independence Blue Cross

Eugean Jiwanmall’s past professional experiences include basic science and clinical research. In his current role as a Senior Research Analyst for Medical Policy & Technology Evaluation at Independence Blue Cross’ Medical & Claim Payment Policy Department within Facilitated Health Networks and Medical Affairs, he has served as the subject-matter-expert, writer, reviewer, communicator, presenter, consultant, adviser/facilitator, and decision-maker for hundreds of medical policy topics and technology assessments within dozens of clinical disciplines and multiple business areas.  Eugean has participated in numerous roles in a number of conferences and summits etc. upon invitation.

Eugean is finishing graduate work in bioethics at the University of Pennsylvania School of Medicine. He holds a bachelor’s degree in biological sciences with honors from Drexel University (minor was in business administration), a master’s degree of public health in evaluative clinical sciences from Dartmouth Medical School, and an MBA in healthcare management and economics with honors from Drexel University.

Dr. Fatima Sulaiman

Head of Research and Services, Scleroderma & Raynaud's UK

Fatima Ann Sulaiman is the Head of Research and Services at Scleroderma and Raynaud’s UK (SRUK). SRUK is a patient focused organisation which funds research into scleroderma, a rare orphan auto immune condition. She is leading on the development and implementation of an innovative research strategy which focuses on working collaboratively across industry in order to deliver benefits to patients sooner. Key areas of focus include precision medicine and advancing orphan drug development which represent large unmet needs for rare disease patients.

Previously, she was a senior manager at Cancer Research UK, one of the largest funders of scientific research in the UK. Prior to this, she completed a PhD from the National Institute for Medical Research (now part of the Francis Crick Institute).

Joanne M. Hackett

Chief Commercial Officer, Genomics England

Joanne M. Hackett is the Chief Commercial Officer at Genomics England, where she is responsible for their industry engagement strategy by developing, managing and accelerating relationships with commercial organisations − creating opportunities for collaboration both nationally and globally.

Professor Hackett is a clinical academic, an investor, and a strategic, creative visionair with global experience spanning successful start-ups to Fortune 500 companies. Aside from her curious passion for life and positivity, Joanne is known for building innovation, driving personalised medicine and leading through fast paced, complex changing ecosystems and integrations. Joanne’s goal is to contribute in bringing the world novel, cost effective and simple health care solutions, and she is particularly keen on building the case for prevention, open science and citizen genomics. She has extensive global experience across academic, business and clinical institutions, and enjoys sharing her experiences with the Boards she sits on as well as companies she provides strategic advice to.

Joanne has been publicly recognised for her relentless pursuit of revolutionising healthcare and has been named the Accenture Life Science Leader of the year 2019, Freshfields Top 100 Most Influential Women 2019, One HealthTech 2018 Top 70 Women in the NHS, Pharmaceutical Market Europe’s  2018 30 women leaders in UK healthcare and BioBeat 2017 Top 50 Women in Biotech Award. Joanne believes in human courage and perseverance against the odds, and demonstrates that positive change, whether in a company or in one’s personal life, can be carved out from even the greatest of trials. As a believer of ‘health = wealth’, Joanne is an internationally known yoga instructor.

Janet Allen

Director of Strategic Innovation, Cystic Fibrosis Trust

Janet Allen trained as a physician and practised in academic medical centres.  Following post-doctoral training at Harvard Medical School, Dr Allen gained faculty positions at the University of Cambridge and was then appointed Chair of Molecular Medicine at University of Glasgow.  Dr Allen moved to senior management posts in Parke Davis and then Pfizer.  Since 2002, Dr Allen has been involved in developing research strategies for the Conway Institute at University College Dublin and for the Biotechnology and Biological Sciences Research Council.  Since 2012, Dr Allen joined the Cystic Fibrosis Trust where she now holds the post of Director of Strategic Innovation. In this role, she has been exploring the use of advanced analytical tools to develop predictive tools for precision/personalised medicine for people with CF.

Thomas Kühler

Head Regulatory Science & Policy, EU/AMEE, Sanofi

Thomas Kühler is a PhD chemist by training with a post-doctoral degree obtained with late Nobel Prize Laureate Donald J. Cram. In recognition of his contributions to the field of Medicinal Chemistry and longstanding experience in drug discovery he was appointed Associate Professor in Medicinal Chemistry at Uppsala University in Sweden.
Thomas has garnered his work experience from Hässle AB in Sweden (now AstraZeneca), the Medical Products Agency (the Swedish drug regulator), Novo Nordisk A/S in Denmark, and most recently Sanofi in France.
Thomas has served on the Board of the Drug Information Association in the US. He also was a member and chair of the Board of The Organisation for Professionals in Regulatory Affairs in the UK. He is currently serving on the Board of the MedTech & Pharma Platform headquartered in Switzerland.

Sheela Upadhyaya

Associate Director - Highly Specialised Technologies, NICE

Sheela Upadhyaya is currently the Associate Director of the Highly Specialised Technology program at NICE and is responsible for running the program to evaluate medicines and technologies for rare and ultra-rare conditions for commissioning in the NHS.

She has extensive experience in understanding the issues that face companies and other stakeholders when trying to secure access for medicines for the orphan and ultra-orphan conditions. These include developing innovative access arrangements in liaison with industry, clinicians, patients and the NHS.


Sheela has co-authored several papers that discuss HTA methods for assessing value of orphan medicines and presented at many conference issue panels on the subject.
Prior to joining NICE, she commissioned rare and ultra-orphan disease services in the NHS delivering improvements through collaborating with industry, clinicians and patient groups. During that time, she successfully decommissioned failing services and seamlessly transferred the care of patients to better quality services.

Sheela has a passion for partnership working and believes that collaboration across the sector is the key to delivering high quality outcomes for all.

Sonali Chopra

Director, Alliance and Advocacy Relations, Genentech, Inc.

Sonali Chopra is the Director of Alliance and Advocacy Relations at Genentech.  In her role, she leads a team responsible for advocacy relations for ophthalmology, neuroscience, immunology, and rare disease.  Previously, she oversaw all of oncology advocacy relations for Genentech.  She joined Genentech as a Senior Manager in Advocacy Relations in January 2010 focusing on support for the breast cancer advocacy community as well as for colorectal cancer, skin cancer, prostate, gastric and ovarian cancer.  Sonali comes to Genentech from Bayer and where she worked in healthcare reimbursement, advocacy relations and public health.  At Bayer, Sonali managed reimbursement and patient assistance for specialty pharma and worked with patient advocates on access-related issues.  Prior to Bayer, she led a project with the non-profit Lesotho-Boston Health Alliance that rebuilt the organizational structure of two pilot hospitals in Lesotho, Africa, and laid the groundwork for a grant to study cervical cancer. She has also worked as a healthcare consultant for Lash Group advising clients on reimbursement launch strategies.  Sonali holds Masters degrees in Business Administration and Public Health from Boston University, and a Bachelor’s degree in Integrative Biology from the University of California Berkeley.

Femida Gwadry-Sridhar

CEO & Founder Pulse Infoframe Inc.

Dr. Femida Gwadry‐Sridhar is the Founder and CEO of Pulse Infoframe Inc. She has her PhD in Research Methodology, Health Economics, McMaster University. She is a pharmacist, epidemiologist and methodologist with over 25 years of experience in clinical trials, disease registries, knowledge translation, health analytics and clinical disease outcomes. She founded the first knowledge translation health informatics lab in North America in 2006, creating a collaborative ecosystem for interdisciplinary research. In 2008 this facility, I‐THINK research, developed a physical and virtual platform to support multidisciplinary research. The platform has evolved to enable the integration of clinical, imaging and histopathology data.

 

Pulse Infoframe has developed healthieTM, a revolutionary evidence-based platform transforming rare disease and cancer clinical research and treatment. Pulse’s SaaS data platform is becoming the de facto standard for real work evidence, meeting an urgent regulatory requirement. Pulse builds multi-stakeholder collaborative networks of pharmaceutical companies, researchers and patients and enables stakeholder access to clinical trials, input on trial design, new treatment development, and accelerated research. Pulse captures, organizes, analyzes, curates and shares existing and unique data using its industry leading and scalable data-sharing platform powered by AI and machine learning.

 

Under Dr. Gwadry-Sridhar’s guidance Pulse has developed a global presence.  Pulse has collaborated with international funding agencies advancing the requirements for rare disease registries. Collecting relevant and actionable data in rare disease is essential.  Pulse has addressed relevant questions about data sharing, governance, ontologies and patient reported outcomes- all key to understanding and enabling the development of real-world evidence. DrGwadry-Sridhar is a life-long collaborator and has done so across different disciplines and cultures. This experience has resulted in a comprehensive understanding of the value of collaboration to fast-forward progress and improve lives everywhere.

Leslie Galloway

Expert Adviser, NICE, Chairman, Ethical Medicines Industry Group

Leslie built a successful career over 40 years in senior management roles in both the pharmaceutical and medical device industries, culminating in the successful turnaround of a pharmaceutical business.

He was elected EMIG Chairman in 2005 and has been instrumental in the metamorphosis of EMIG from being a business/networking group to the influential trade association it is today. In 2005, EMIG began with 17 Member Companies and today, has 270+, of which, 90% choose to be represented only by EMIG. Consequently, EMIG represents c. 50% of UK branded medicines.

In July 2014, Leslie was elected VP, European Confederation of Pharmaceutical Entrepreneurs (EUCOPE). He was Vice Chairman of the NICE Project Development Group for Local Formularies Good Practice Guidance, a member of the NICE Medicines Optimisation Guidance Development Group and was recently appointed as an Expert Adviser to the NICE Centre for Clinical Practice.

He was a member of the Accelerated Access Review Reference Group and a member of the Cancer Drugs Fund Working Group. He has published numerous articles, is a frequent speaker at national and international seminars, a Member of the Scottish Government’s Strategic Engagement Group, a Member of MENSA and is a visiting lecturer at the University of Cambridge.

Dr. Nicolas Sireau

CEO and Chair of Trustees, AKU Society

Dr Nicolas Sireau is the CEO and Chair of Trustees at the AKU Society, a patient group that helps people with AKU (short for alkaptonuria), a rare genetic disease affecting both his children. He is also co-founder and Chair of Findacure, an organisation that helps rare disease patient groups. Previously, Nick was the CEO of SolarAid, an NGO working in Africa. He is a fellow of the Ashoka Fellowship of Social Entrepreneurs and has a PhD in the social psychology of social movements. He is the editor of ‘Rare Diseases: Challenges and Opportunities for Social Entrepreneurs’ (Greenleaf 2013) and of the ‘Patient Group Handbook: A Practical Guide for Research and Drug Development’ (Findacure 2016).

Dr. Linda Popplewell

Department of Biological Sciences, Royal Holloway University of London

Expertise lies in the development of pre-clinical gene therapies for neuromuscular diseases, in particular muscular dystrophies based on gene addition, endonuclease-mediated gene editing, antisense oligonucleotide (AO)-induced exon skipping and gene silencing. Having performed well in clinical trial, FDA approval is currently being sought for a patented AO developed in the Popplewell lab for a particular subset of Duchenne muscular dystrophy (DMD) patients. Dr Popplewell is PI of an upcoming clinical trial of an optimised microdystrophin AAV, which would have universal DMD patient applicability. She is an elected board member of the BSGCT, sits on the Management Committee of an eCOST Action and is an invited member of the MDEX Consortium.

Martina Garau

Director, OHE

Martina is a Principal Economist at the Office of Health Economics, which provides authoritative resources, research and analyses in health economics, health policy and health statistics. She has over 10 years of experience managing and leading projects for a variety of organisations, including global pharmaceutical and life sciences companies, non-profit research organisations, charities and trade associations.

Her current research includes methods and applications of Multi-Criteria Decision Analysis (MCDA) approaches in healthcare decision making; and economic issues posed by the development and provision of treatments for rare diseases. She has also worked on other topics related to health policy research around: priority setting and Health Technology Assessment (HTA) processes in middle-income countries; the economics of personalised medicine for health care payers and the pharmaceutical industry; the application of HTA methods for the assessment of interventions in broad  therapy areas, such as cancer and orphan medicines.

Martina joined the Office of Health Economics in 2004, following a placement at NICE in London. She has an MSc in Economics from the University of York and a first degree in economics from the University of Cagliari in Italy.

Karen Facey

Sr. Research Fellow, Impact HTA - Co-Lead Investigator, WP10

After working as a senior statistician in Pharma and UK medicines regulation, Karen Facey became the founding Chief Executive of the first national health technology assessment (HTA) agency in Scotland. For the past 17 years, Karen has been an independent consultant on health policy, HTA and patient involvement.  She has been a Non-Executive Director on a Scottish Health Board and for 11 years led work on the NHSScotland resource allocation formula. She now has a part-time role as Co-Principal Investigator in the EC H2020 funded IMPACT HTA project at the University of Edinburgh. She is leading a work package on appraisal of rare disease treatments, which has involved review of international appraisal processes, ethnographic observation of committees and development of principles for outcomes-based managed entry agreements. Much of this work has involved review of processes for ATMPs, with specific work undertaken on the potential use of RWE to support access.

Robert Johnstone

Patient Advocate - International Foundation for Integrated Care (IFIC), European Forum for Good Clinical Practice (EFGCP) & Health Quality Improvement Partnership (HQIP)

Chair: Access Matters, Midstream, Board Member: European Forum for Good Clinical Practice (EFGCP), Health Quality Improvement Partnership (HQIP), International Foundation for Integrated Care (IFIC). Having had Rheumatoid Arthritis for nearly 60 years from the age of three, I have a wealth of experience as a user of health services. Since 1990 I have voluntarily represented people with disabilities & chronic conditions locally, nationally & internationally “The people have the right & duty to participate individually & collectively in the planning & implementation of their healthcare” Alma Ata Declaration – Principle IV, 1978 World Health Organisation.

Karen Matthews

Business Development Manager, NIHR

The National Institute for Health Research (NIHR) Clinical Research Network works in partnership with the National Health Service (NHS) to deliver high quality research. We support all phases of clinical research studies and trials across all disease areas.

Dr Karen Matthews facilitates discussions between non-commercial funders of research, such as charities, and the Clinical Research Network. She provides advice to both charities and companies on how they are able to collaborate with the Clinical Research Network. Karen also provides guidance on how to access and optimise our Study Support Service to ensure that clinical studies are set up efficiently and deliver to their recruitment targets.

Karen has been with the Clinical Research Network since December 2007. She was previously the operational lead for the Coordinated System for gaining NHS Permission (CSP). The system, which has now been superseded, was instrumental in demonstrating that the UK could set up clinical trials rapidly in a coordinated manner.

Karen has previously worked as a research scientist within the oncology team in Glaxo Wellcomne (1995-6). She gained a PhD in developmental genetics (2000) and conducted post-doctoral research within the Institute of Child Health, London. She subsequently went into research administration and has held posts with Cancer Research UK and Barts and The London NHS Trust.

Paolo Morgese

EU Director Market Access, Alliance for Regenerative Medicines

Paolo is a healthcare investment and market access professional with more than 15 years of experience in both assessing and supporting access to innovative healthcare technologies. Paolo is currently VP Market Access at Alira Health an healthcare advisory company and EU Director Market Access and Member Relations at the Alliance for Regenerative Medicine (ARM). Between 2012 and 2017 he worked at Deerfield Management, a healthcare investment company, where he was Director of EU Research and Market Access. Prior to that, he worked at Merck Serono for five years, Kyphon, which became Medtronic, and also spent several years with the Italian HTA Agency Agenas. Paolo’s interests and expertise are centered around healthcare technology value assessment and maximization, with a particular interest in the early stages of product development.

Emily Crossley

Co-founder and joint CEO, Duchenne UK

Emily set up the Duchenne Children’s Trust in 2012 after her son was diagnosed with Duchenne. Before that she was a reporter and anchor for Channel 4 News and CNN International. The Duchenne Children’s Trust raised £3.5million in its first three years, to spend on clinical trials, research, and clinical trial infrastructure. The charity set up the annual Duchenne Dash, which brings in money for research and raises awareness around Duchenne muscular dystrophy. Emily has advocated on behalf of patients around the world. She’s spoken many times in the Houses of Parliament. She is a member of the MHRA patient group consultative forum.

Rafał Świerzewski

President of Board, Fundation for Patients with Rare Diseases COCARDA

Rafal Swierzewski has been involved in patient advocacy for ten years as both a scientist and rare cancer survivor. He earned his PhD in 2006 in chemistry by examining physical and chemical properties of model macromolecules (incl. new chemotherapy compounds) in cellular environment. From 2013 to 2016 he served as Board Member of the European Cancer Patient Coalition (ECPC). Currently, he continue his activity, representing ECPC in Patients’ and Consumers’ Working Party within the European Medicines Agency (EMA). Through the EMA, he is involved in various projects, such as Adaptive Pathways, IMI-ADVANCE (Scientific Advisory Board member) and IMI-WEBRADR. Rafal also represents patients’ interests in the Stakeholders Group for European Union’s clinical trial portal and database, created by EMA to construct new system for implementing European regulations on clinical trials and clinical data. Rafal is also a member of Technical Anonymization Group (TAG) of the EMA, which continue the work undertaken during the development and finalisation of the external guidance on the anonymization of clinical reports for publication. From March 2018 he is President of the Board of Foundation for Patients with Rare Diseases “COCARDA”, an innovative patients’ driven research organization established in Poland.

Jim Green

Chairman of Board of Trustees of INPDR (International Niemann-Pick Disease Registry).

Jim had two sons diagnosed with Niemann-Pick type C disease in the early 1990’s.

Jim is a retired head teacher who, in 1991, founded the Niemann-Pick Disease Group (UK). He was Chairman of that organisation until 2012. Jim was also an original member of the NNPDF (National Niemann-Pick Disease Foundation) board when it was formed in the USA in 1992. He continued to serve on that board until 2004. In the twenty-eight years since 1991 Jim has witnessed many advances in research and understanding of NP issues. He has met many patients, researchers, clinicians and other people involved with these diseases. In 1991 there were no support foundations and little or no information. Jim has seen the growth of parent involvement and associations’ influence and has been witness to the huge difference people can make when they work together. In October 2009 this took another step forward with a collaborative venture between the NNPDF and the NPUK when they brought together Niemann Pick Foundations from around the world to network and share information. This resulted in the formation of the “International Niemann-Pick Disease Alliance” – the INPDA. Twenty organisations are now members. Jim was President of the INPDA until June 2019. The project to build an international NP disease registry (INPDR) was started in 2013 and the first development phase was completed in September 2016.  It has steadily developed and grown since then and is now attracting much interest in the rare disease community.