April 25 - 27, 2018
Gaylord National Harbor Hotel, Oxon Hill, MD
World Orphan Drug Congress USA focusses on the most pressing challenges and opportunities to bring rare disease therapies to patients faster.
World Orphan Drug Congress USA has evolved from a one track conference focused on regulatory pathways, policy and incentives, to a global gathering of 1,000 leaders in orphan drugs from 38 countries, and over 135 presentations covering all aspects of orphan drug development and rare disease research.
Enjoy discussions on:
Cell & Gene Therapies
Genetic Testing
Gene Editing
Partnering & Investment
Global Market Access
Expanded Access Programs
Real World Evidence
Global Patient Advocacy
Value based pricing & reimbursement
Epidemiology & forecasting
Orphan Drugs Policy
Gene Therapy Development
Edmund Jessop has been practising public health for the NHS in England since 1981 in various jobs at district, regional and national levels. Since 2002, he has been medical adviser to the team which plans, funds and monitors services for patients with very rare disease. This has included decision...
Katri Asikainen, Chairperson, Finnish Rare Diseases and Disabilities Organization (HARSO)Katri Asikainen is the Chairperson for the Finnish Rare Diseases and Disabilities Organization (HARSO). Her background and expertise in IT has been invaluable in the coming age of digitalized healthcare system...
Denis Belyakov CEO of Russian Union of patients’ and patients’ organizations. Denis leads the cross-functional, umbrella, interregional patient’s organization responsible for patients’ support and access to treatment for different rare diseases. This includes not only the end-result of successful...
Mr. Ingram was appointed our President and Chief Executive Officer and a member of our Board of Directors in June 2017. Prior to his appointment as Sarepta’s President and Chief Executive Officer, Mr. Ingram served as the Chief Executive Officer and President and a Director of Chase Pharmaceuticals ...
Beata Ferencz, Project Manager, Rare Diseases Sweden Beata Ferencz is a project manager at Rare Diseases Sweden, an umbrella organization that represents rare diseases patients in Sweden. Her current project “Learning together – person centered care for rare diseases “has the primary aim to improve ...
STEPHEN FRANKLIN, CHIEF EXECUTIVE OFFICER Stephen, the founder of Evgen Pharma, has over 20 years’ commercial experience in life science industries, focusing on the commercialisation of new technology. He was the founder of Provexis plc, a nutraceutical company, and was its chief executive at the...
Jayne C. Gershkowitz joined Amicus in June 2006 and currently serves as Senior Vice President and Chief Patient Advocate. She is a long-time patient advocacy professional with expertise in the rare disease community, specifically in the LSD space. Prior to joining Amicus, Ms. Gershkowitz served as...
Birthe Byskov Holm, President, Rare Diseases DenmarkBirthe has been a volunteer advocate for people with rare diseases and their families since her son was born i 1983 with a rare disease.She is one of the cofounders of the Danish Alliance for rare disease patient organizations – Sjaeldne Diagnoser ...
Dr. Kaminsky joined the International Rett Syndrome Foundation in January of 2012. He came to the Foundation after serving 11 years as the Vice President of Research for the Uniformed Services of the Health Sciences, the Department of Defense Medical School serving all the uniformed services of the ...
Igor Lokot, born 1972, Member of the Board in Double Bond Pharmaceutical International ABsince April 2015. Igor has a PhD in Bio-organic Chemistry and a wide experience in drug development. Igor is one of the founders of the pharmaceutical company Oasmia Pharmaceutical AB which is listed on NASDAQ...
Dr. Padilla is Professor of Pediatrics at the College of Medicine and currently Chancellor of University of the Philippines Manila (www.upm.edu.ph). Recognizing her varied contributions to the academic growth of genetics in the Philippines, she was conferred Academician of the National Academy of...
Dr Alan Robertson holds a PhD in synthetic organic chemistry from the University of Glasgow. Following a post doctoral appointment with the Nobel Laureate, Sir John Cornforth, he took a position as a research scientist with Wellcome plc (now part of the pharmaceutical company GSK) working on drug...
Mr. Christer Rosen, Founder, serves as the Chairman of the Board and Chief Executive Officer at Jupiter Orphan Therapeutics, Inc. Mr. Rosen is the Founder of EffRx, Inc., serves as the Chief Executive Officer and Treasurer. Mr. Rosen served as an Executive Vice President of Top Source Technologies...
Dr. Tenaerts works closely with the CTTI Executive Committee to develop and implement strategies to accomplish CTTI’s mission. She provides senior level oversight of the day-to-day operations of CTTI and orchestrates efforts to effectively engage all interested stakeholders to improve the conduct...
Martin WelschofChief Executive Officer, Opsona TherapeuticsBefore joining Opsona in Feb 2012, Martin was the Managing Director and Co-founder of Affitech A/S, a biopharmaceutical company focused on the discovery and development of human antibodies. Under his leadership Affitech AS was transformed...
Your customers are here continuously looking for new strategies and technologies to expedite orphan drugs to patients.
Find out how World Orphan Drug Congress USA can solve all your sales and marketing needs.
AccessPharmaCon,LLC Aegerion Pharmaceuticals Aetna Pharmacy Management Alexion Pharmaceuticals Andarix Pharmaceuticals ArmaGen Technologies, Inc. Ashfield Healthcare Asklepion Pharmaceuticals LLC Ax-S Pharma B.D.C. Venture Capital BioBlast Pharma Ltd Biomarin Pharmaceutical Inc. BioPontis Alliance for Rare Disease Brace Pharma Capital Brazilian Federal Council of Medicine Bristol-Myers Squibb CatalentPharma Solutions CatalystPharmaceutical Partners CDER FDA Clear Pharma Clementia Pharmaceuticals Inc. Clinuvel Pharmaceuticals Ltd Dicerna Pharmaceuticals DrugDev EvaluatePharma Ltd Genoa Pharmaceuticals Genpharm GenSight Biologics Genzyme Corp Giddi Pharma Co., Ltd. Idera Pharmeceuticals IDIS Pharma Iptechex Pharmalicensing Jazz Pharmaceuticals, Inc Juice Pharma Worldwide KBI Biopharma Inc Lumos Pharma M3 Pharma Marathon Pharmaceuticals LLC Marinus Pharmaceuticals Momenta Pharmaceuticals Inc Opsona Therapeutics Ltd Optio Biopharma Solutions LLC Orpharma Pfizer, Inc Pharmaceutics International In Pharmaessentia Corp Pharnext Portola Pharmaceuticals , Inc. Provectus PharmaceuticalsInc QuayPharma Quintiles Inc Raptor Pharmaceuticals Corp Reata Pharmaceuticals Recordati S.p.A Recursion Pharmaceuticals Retrophin Sanofi Savara Pharmaceuticals Seattle Children's Research Institute Shire Pharmaceuticals Inc Taiba Pharma LLC Teva Threshold Pharmaceuticals Inc Tolero Pharmaceuticals Ultragenyx Pharmaceutical Inc. Valley Fever Solutions Vanda Pharmaceuticals Inc Vertex Pharmaceuticals