The 2nd annual World Orphan Drug Congress showcases drug development, new drug discovery and innovations related to rare diseases.      
 

Karen Aiach is founding president and CEO of LYSOGENE, a clinical stage biotechnology company specialized in intracerebral gene delivery for the treatment of neurological diseases. In less than five years, Karen brought LYSOGENE’s first product SAF-301 into the...

Mr. Aldag joined AMT in October 2009 as Chief Executive Officer. He has twenty nine years of experience in executive, business and financial management positions at Evotec AG, MAN AG and Treuhandanstalt. Over 11 years initially as CFO and then as CEO, he was...

Dr. Aviezer has served as Chief Executive Officer of Protalix BioTherapeutics (NYSE:PLX) since 2002 and since 2005 he serves also as President and member of the Board of directors. Dr. Aviezer has 15 years of experience in senior biotechnology management, advancing...

Ségolène Aymé is a medical geneticist, Director of research at the French Institute of Health and Medical Research (INSERM). She is the executive manager of the INSERM department dedicated to information on rare diseases in Paris. This service established Orphanet...

Dr. Benichou has 18 years of experience in the management of clinical development of drugs and high-tech medical devices in Europe and in the USA. After she earned a medical degree from the University of Toulouse in France, she obtained inter-university degrees in...

Dr Catherine Berens was trained as a pharmacist and obtained her PhD in Pharmaceutical Sciences from the University of Louvain in Belgium. After having worked for the European Directorate for the Quality of Medicines (European Pharmacopoeia, Council of...

Kyle is the founder and CEO of PatientCrossroads, a provider of patient registry solutions to the rare and neglected disease community. PatientCrossroads collects patient provided medical history and testing results in order to gain insight into disease progression...

Mark Corbett is the Global Strategic Planning Director at Clinigen

Remco de Vrueh currently works for the Netherlands Organisation for Health Research and Development (largest Dutch health research funding agency) as advisor to various (orphan) drug and innovation programmes. He is currently involved in two projects. The first project ...

Alexandre Delacoux is European Biophamaceutical Enterprises (EBE) Executive Director since January 2011. With over 20 years professional experience in European and international public affairs, Alexandre Delacoux brings to EBE a unique combination of experience in the...

Robert Derham blends over 16 years of business (biotech/pharma) and research experience, which is needed to manage CheckOrphan and understand the evolving field of rare diseases. In the past, he has successfully launched interactive, life science-based platforms for...

Catarina Edfjäll, Ph.D., joined Shire HGT as VP, EU and International Regulatory Affairs in June 2011. She has 15 years of experience in global regulatory affairs, and has worked with both innovative biotech products and small molecules at all product life cycle...

An economist by training, Richard came to the UK in 1998 to study for a PhD. His thesis considered how the views of the public could be used to inform public policy decisions, considering both the elicitation of such preferences and a possible interpretation within...

Chris joined AVI as President and Chief Executive Officer on January 1, 2011. He has served as a director of the Company since June 2010. Previously he was Vice President of Corporate Strategy for Celgene Corporation from July 2007. From November 2005 to June...

Josie Godfrey is Head of Policy for the National Specialised Commissioning Team in England. She led work to establish the new Advisory Group for National Specialised Services (AGNSS) that makes recommendations on which services and technologies for people with very...

Cláudia Hirawat is responsible for Business and Corporate Development spearheading PTC’s collaborations. Ms. Hirawat joined PTC in 2000 as the company's seventh employee. Partnering with the CEO and the senior management team, Ms. Hirawat played a key role in building...

Wills Hughes-Wilson is Vice President, Health Policy Europe at Genzyme. She joined Genzyme in September 2005. In this function, Hughes-Wilson is responsible for public policy and public affairs, including activities related to market access and reimbursed patient...

Dr. Incerti serves as Officer of Genzyme Corporation, where he oversees Global Medical Affairs , which includes Medical Operations, Global Regulatory Affairs and Compliance, Corporate Quality Systems, Global Patient Safety and Risk Management, Evidence Value...

Alastair Kent is the Director of Genetic Alliance UK – the national charity of over 140 patient organisations, supporting all those affected by genetic conditions. Genetic Alliance UK’s mission is to promote the development of the scientific understanding of genetics...

Craig has over 25 years of experience in the healthcare sector. He is a respiratory therapist and has worked in hospital settings as a clinician and administrator. His prior corporate affiliations include Quantum Health Resources, Inc. where he held management...

• Wytske Kingma joined Genzyme in 1993. She created a global safety-and in 1998 a medical information department, and built it into the global organization it is today, with 100+employees in US and many more in various locations around the world. The PV/MI team is...

Dr Deborah Marsden is a graduate of the University of New South Wales in Sydney, Australia, where she completed early residency in general medicine and then pediatrics. She completed further pediatric training in London, UK and at the University of California at San...

Susan McGoldrick is the CEO of QCTR Ltd, a CRO specialising in CNS and orphan diseases. QCTR recently completed one of the largest phase III clinical trials in Huntington's disease, recruiting over 430 patients in 12 months. Susan is an experienced pharmaceutical...

David P. Meeker is responsible for Genzyme’s commercial organization, overseeing the business units, country management organization and global market access functions. He works to maximize the potential of the company’s broad portfolio of products, most of which are...

Dr. Meier is Chief Scientific Officer of Santhera Pharmaceuticals (SIX: SANN), a specialty pharmaceuticals company focusing on the research, development and commercialization of innovative therapies for the treatment of orphan neuromuscular diseases. Santhera is based...

Sue Millman is the Chief Executive of medical research charity Ataxia UK. It is estimated that 10,000 people in UK have ataxia, a degenerative neurological condition which is often inherited, and for which there is currently no cure. She is also Secretary General of...

Philippe Monteyne, MD, PhD is Senior Vice President - Head of Development and Chief Medical Officer, GSK Rare Diseases. He reports to Marc Dunoyer, GSK's Chairman Japan, who is leading the new GSK special unit created in 2010. This unit is specialised in the...

Peter Myrenfors has worked in the field of Orphan drugs and rare diseases for the last four years. He presently holds a position as Director of Medical Affairs in the newly formed Swedish Orphan Biovitrum, Stockholm Sweden and prior to the merger Dr Myrenfors was the...

Dr. Old is Senior Advisor and Acting Scientific Deputy Director of the NIH Center for Translational Therapeutics (NCTT) at the National Institutes of Health. The NCTT, which includes the NIH Chemical Genomics Center (NCGC), the Therapeutics for Rare and Neglected...

Francis Pang is the Senior Market Access and Public Affairs Director for Shire HGT

Larry Park manages and directs drug discovery campaigns from Lead Optimization through IND filing. Prior to CHDI, Larry was a Senior Research Scientist of Vertex Pharmaceuticals, Inc., where he led the CNS In vivo Pharmacology group in developing small molecule kinase...

Hear Samantha Parker speak at the World Orphan Drug Congress 2011

Born in Rome, Andrea Rappagliosi received a law degree from the University of Rome “La Sapienza” and completed a post-graduate Masters at the Istituto Superiore dei Studi Legislativi (ISLE) from which he obtained a “Legislative Consultant Diploma”. He began his...

Frédéric Revah joined Genethon as CEO in January 2010 and bringing over 20 years' experience in the pharmaceutical and biotech industries as well as academic research. Immediately prior to joining Genethon in January 2010, he had been Chief Executive Officer of the...

Charlie Richard is Principal Medical Director and Head of Translational Medicine at Shire Human Genetic Therapies in Lexington, Massachusetts, USA. At Shire, Charlie directs a team of physicians overseeing early-stage CNS enzyme replacement therapy clinical...

Reporting to Marc Dunoyer, Global Head of GSK Rare Diseases, Pierrick oversees global market access, advocacy and communication strategy for GSK Rare diseases. Dual education in International Business & Economy (MBA IMD Lausanne, MA international Economy Paris...

Mark Rothera is responsible for the commercial operations of Shire Human Genetic Therapies in Europe, the Middle East & Africa. Mark joined Shire in early 2006 just after the acquisition of TKT and since then has been instrumental in driving a step change in growth in...

Hans Schikan is CEO of Prosensa, an innovative Dutch biopharmaceutical company focusing on rare diseases like Duchenne muscular dystrophy, using its RNA modulation platform. Prior to Prosensa, Hans worked at Genzyme as VP Global Marketing and Strategic Development for...

Evonne Sepsis has spent the last 18 years in the healthcare sector and has been involved in raising over $3.5 billion for life sciences companies. In 2009, Ms. Sepsis founded ESC Advisors. Prior to ESC, Ms. Sepsis was the head of healthcare investment banking at...

Dr. Shih joined Pfizer’s Orphan and Genetic Disease (OGD) Research Unit as the unit was forming in early 2010. Alvin applies his clinical and business experience to a variety of strategic and managerial roles within the group. Prior to joining Pfizer, Alvin was a...

Dr Nicolas Sireau is Chairman of the AKU Society, a medical charity that works to find a cure for and support patients with AKU, which affects his two sons. AKU (short for Alkaptonuria) was the first metabolic disease ever identified, in 1901 in London. It is a...

Sharon F. Terry is President and CEO of the Genetic Alliance, a network transforming health by promoting openness as process and product, centered on the health of individuals, families and communities. She is the founding CEO of PXE International, a research advocacy ...

An van Es-Johansson is Head of Clinical Development at Swedish Orphan Biovitrum ,which she joined in 2006. An started her career in the pharmaceutical industry more than 20 years ago for Eli Lilly in the Netherlands, working as a Medical Director on rhGH in Turner’s...

Since 1995 I have been a person living with Behcet’s Syndrome, which is a rare illness. Behcets is an inflammation of the capillaries and can affect many parts of the body and crosses many fields of medicine. There is no cure and no diagnostic test for the illness. It...

Jerome B. Zeldis is CEO of Celgene Global Health and Chief Medical Officer of Celgene Corporation, Summit, NJ. Prior to that he was Celgene’s Senior Vice President of Clinical Research and Medical Affairs and has been at Celgene since February, 1997. He attended...

Martine Zimmermann is currently Executive Director for Global Regulatory Affairs at Alexion Pharma International. She is based In Lausanne Switzerland. Dr Zimmermann has gained extensive experience in international pharmaceutical industry for more than 18 years in...