The 8 ^th Global Orphan Drug Conference and Expo

Jacqueline Corrigan-Curay, J.D., M.D., serves as Director of CDER’s Office of Medical Policy (OMP). She leads the development, coordination, and implementation of medical policy programs and strategic initiatives. She works collaboratively with other CDER program areas, FDA centers, and...

Mark brings 28 years of experience in the bio-pharmaceutical industry including driving the transition of multiple emerging biotechnology companies from R&D stage to commercialisation. During his career, he has focused on bringing novel therapies to market for patients with rare diseases, and...

Dr. Sandy Macrae serves as Sangamo’s President and CEO and as a member of the Board of Directors. He has twenty years of experience in the pharmaceutical industry previously serving as the Global Medical Officer of Takeda Pharmaceuticals, from 2012 to March 2016, where he established and led the...

As Director of Health Economics at ICER, Rick directs the development of economic evaluations to accompany rigorous reviews of clinical evidence, assessing the potential costs, cost-effectiveness, and budgetary impact of a wide range of diagnostic, prognostic, therapeutic and other clinical...

Yann Le Cam is a patient advocate who has dedicated 25 years of professional and personal commitment to health and medical research non-governmental organisations in France, Europe and the United States in the fields of cancer, HIV/AIDS and rare diseases.He has three daughters, the...

Mr. Ronald Bartek is FARA's Co-founder and President; Chairman of the Board of the National Organization for Rare Disorders (NORD); 4-year member, NIH National Advisory Neurological Disorders and Stroke Council, and former partner and president of a business and technology...

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...

Saira BatesCo-founder & CEO, Escend PharmaceuticalsSaira started her research career at Fox Chase Cancer Center and later held positions at Sterling Drug, Syntex, and Matrix Pharmaceuticals. Saira has provided preclinical support to several oncology programs, two of which are now marketed...

Carole Ben-Maimon, MD joined Chondrial Therapeutics in December 2016 as the Chief Executive Officer, bringing more than 25 years experience in the pharmaceutical industry to the company. Most recently, she worked with Deerfield Management Company assisting in the evaluation of investment...

Dr. Bhat founded Reviva Pharmaceuticals (Reviva) in 2006 and since its inception, Reviva has advanced extremely rapidly under his leadership, with a portfolio of proprietary compounds at different stages in a pipeline encompassing chronic diseases in large therapeutic areas such as...

Dr. Preston Campbell is the president and chief executive officer of the Cystic Fibrosis Foundation. He previously served as the Foundation's executive vice president for medical affairs. Dr. Campbell has more than 25 years of experience caring for people with CF. Most recently, he oversaw the...

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...

Dr. Olivier Danos, Ph.D. has been Chief Scientific Officer of Regenxbio Inc. since March 2017. Dr. Danos serves as the Senior Scientific Advisor of the entire SAF-301 program. He served as Senior Vice President of Cell & Gene Therapy at Biogen Inc. until March 2017 and served as its Senior Vice ...

Dr. Rylan Hanks’ expertise is in global regulatory and health policy, with a focus on the United States. He currently serves as a Director of Global Regulatory and R&D Policy at Amgen. Prior to this role, he worked at the U.S. Food and Drug Administration within the Office of Generic...

Dr. Kathy High, an accomplished hematologist with a longstanding interest in gene therapy for genetic disease, began her career studying the molecular basis of blood coagulation and the development of novel therapeutics for the treatment of bleeding disorders. Her pioneering bench-to-bedside...

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 ...

Cristina Whalen Klafehn is the Associate Director of Development Sciences, Patient Advocacy, for the Achondroplasia program at BioMarin. In this role, Cristina aims to identify needs and opportunities to incorporate the patient voice and patient advocacy group perspective across the research and...

Ken Mills is the founding President and Chief Executive Officer of REGENXBIO. Prior to REGENXBIO, Mr. Mills was the Chief Financial Officer and Vice President of Business Development at Meso Scale Diagnostics, a privately held life sciences company. There, he served as a member of the founding...

Lara Sullivan brings to SpringWorks Therapeutics more than two decades of experience and senior leadership in biopharmaceuticals, healthcare and life sciences, most recently at Pfizer.  While at Pfizer she helmed strategy and portfolio operations for the company’s early stage pipeline and led the...

An experienced entrepreneur with a strong scientific and operational background, Sue brings a decade of experience in pharmaceutical management and research with Abbott Labs and Eli Lilly & Company and more than 16 years of senior management experience with entrepreneurial firms in Florida...

Barbara has been involved in patient advocacy activities for patients with rare diseases for the past 15 years at Ucyclyd Pharma and BioMarin. She joined Audentes Therapeutics as Vice President of Patient Advocacy and Medical Affairs in 2013. She has been actively involved with the National...

Founder and involved in the daily operations of cysticfibrosis.com since 1996, Jeanne is a graduate of Fordham University (Lincoln Center) NYC. Jeanne is a teacher by profession, having spent many years teaching high school math and computer science. She is a well-versed patient advocate with a...

Deputy Chair, Compassionate Use Advisory Committee (CompAC), NYU School of Medicine; Deputy Director, NYULMC Working Group on Compassionate Use and Pre-Approval Access; Rudin Postdoctoral Fellow, Division of Medical Ethics, NYU School of Medicine Dr. Bateman-House is a Rudin Postdoctoral...

Dawn is Reata’s Chief Commercial Officer and leads marketing, market access, sales, training, and commercial operations. She joined Reata in September 2016. Prior to joining Reata, Dawn served as Vice President of Sales with Pharmacyclics, LLC, where she built and led their first hematology...

Philip John (P.J.) Brooks joined the NCATS Division of Clinical Innovation (DCI) as a program director in January 2015. He is the lead program director for the Clinical and Translational Science Awards (CTSA) Program Collaborative Innovation Awards, designed to fund projects that will result in...

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...

Pat Furlong is the Founding President and CEO of Parent Project Muscular Dystrophy (PPMD), the largest nonprofit organization in the United States solely focused on Duchenne muscular dystrophy (Duchenne). Their mission is to end Duchenne. They accelerate research, raise their voices in...

Pamela Gavin sets the strategic direction for NORD and oversees the implementation of the programs and services that support the rare disease community. Before NORD, Pam worked to execute healthcare programs aimed at improving safety and delivery. For the federal government, she implemented a...

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...

Scott A. GrayCEO, Clincierge Scott Gray is an acknowledged leader and innovator in the world of medical meetings, patient-centered clinical trial support and healthcare transportation. In 2013, while hearing of patients’ challenges with logistics to participate in a global clinical trial, the...

Phillippe GuedatCEO / CSO, InFlectis BioSciencePhilippe GUEDAT, PhD, InFlectis BioScience CEO/CSO (Founder), has 20 years of drug discovery experience having led drug discovery department at Vivalis (now Valneva, Nantes, France), where he developed anti-HCV molecules and industrialize a...

Kenneth Hobby is President of Cure SMA.  He leads the organization’s work to develop treatments for Spinal Muscular Atrophy, the number one genetic cause of death for infants, by merging an impassioned grassroots mission with a sophisticated industry incentivizing drug development model.Kenneth...

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 ...

Alastair Kent, Ambassador, Genetic Alliance UKAlastair Kent was the Director of the Genetic Alliance UK. This is the alliance of patient support organisations for all those affected by or at risk from genetic disorders. Our aim is to improve the lives of people affected by genetic conditions by...

David Lapidus is the founder of LapidusData LLC, a consulting firm specializing in market validation services for companies developing orphan drugs.  Mr. Lapidus has extensive experience in interpreting epidemiology and market research to create financial models for therapies where underlying...

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...

Jay Madan, Founder & President, Innovate BiopharmaceuticalsMr. Madan founded Innovate Biopharmaceuticals in 2012 and currently serves as President and member of the Board of Directors. Mr. Madan has 25+ years in biotech, life sciences, and IT as a leader in project development, driving emerging ...

Dr. McGrath has over 22 years of clinical research and pharmaceutical business development experience. From 2013 to 2017, he was Vice President, Business Development, and Head of Search & Evaluation at Bristol-Myers Squibb, based in Princeton, NJ. During this period, the business development...

Dr. Missling, President and CEO of Anavex™, has over 20 years of healthcare industry experience within large pharmaceutical companies, the biotech industry and investment banking. Prior to joining Anavex, he served as the Chief Financial Officer of Curis and ImmunoGen. In addition, at Aventis...

Dr. Murphy’s professional career has spanned 25 years and his positions within the pharmaceutical industry emphasize the integration of medical and scientific acumen with operational excellence. He is board-certified in psychiatry and has a doctorate in pharmacology, with training at Tulane...

Francois Nader, MD, is currently the chairman of Acceleron Pharma and a leading value builder in the biopharma industry.  He was President, Chief Executive Officer and Executive Director of NPS Pharma from 2008 to 2015, when the company was acquired by Shire for $5.2B.  During his tenure as...

Yukiko Nishimura is President/founder of NPO ASrid (Advocacy Service for Rare and Intractable Diseases’ stakeholders in Japan). ASrid is very unique organization and committed to providing the valuable service/system for “connecting” and “creating” with/among multi-stakeholders related to its...

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. Harsha Rajasimha has over 15 years of experience and widely recognized in the field of systems biology, Genomics BigData analytics, rare diseases and precision medicine for cancer. Dr. Rajasimha is co-founder and board member of the non-profit organization for rare diseases India...

Trevor joined CADTH in 2010 as a Clinical Research Officer, and was quickly promoted to Clinical Research Manager. While at CADTH, Trevor has expanded his health technology assessment and drug review experience by completing assignments with our health economics and medical device portfolios. ...

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...

Chandler D. Robinson, MD MBA MScCo-Founder and Chief Executive OfficerDr. Robinson, in 2010, co-founded and was CEO of Tactic Pharma. He helped lead Tactic through a successful sale of its lead compound. His passion is leveraging his interests in medicine, science, and finance to help bring new...

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...

Peter L. Saltonstall is the President and CEO of the National Organization for Rare Disorders (NORD). He joined NORD in 2008 after having served for more than 30 years as a senior official in both for-profit and not-for-profit healthcare environments.   Under his leadership, NORD has forged...

Graduated in Business Administration from FAAP (Fundação Armando Álvares Penteado), specializing in creativity and entrepreneurship. He has had his own business and worked for 6 years in the retail and education sectors, when in 2011 Multiple Sclerosis (MS) came into his life. Since then his...

Mr. Schliebner is a clinical scientist by training, with a 20-year background in clinical drug development.  His expertise is focused on providing strategic, scientific and operational guidance to clients on clinical trial design, competitive landscape analysis, investigator selection, advocacy...

Dr. Takigiku is co-founder, President and CEO of Bexion Pharmaceuticals, Inc. Previously, he was at Procter & Gamble Pharmaceuticals, and was also interim co-Director of the Genome Research Institute (now the Reading Campus) of the College of Medicine, University of Cincinnati. Dr. Takigiku’s...

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...

Durhane Wong-Rieger, President & CEO, Canadian Organization for Rare DisordersDURHANE WONG-RIEGER, PHD is Chair of Rare Disease International (the global alliance of rare disease patient organizations), Past-Chair of the International Alliance of Patient Organizations, member of the Editorial...

David Cory is an industry veteran with over 25-years of operating experience leading teams in large pharmaceuticals, emerging stage biotechnology, and specialty pharmaceutical organizations with an established track record across multiple therapeutic areas, including over 10 new prescription...