International Conference on Orphan Drugs and Rare Diseases 2025

Daniel Wainstock is a Rare Disease Patient Advocate and Health Law Researcher at the Pontifical Catholic University of Rio de Janeiro (PUC-Rio), in Brazil. He advises lawmakers and policymakers in Latin American countries on Rare Disease-related policies. He has also worked as a Volunteer at Rare Diseases International (RDI), on advocacy efforts for the WHO Resolution on Rare Diseases.

Dr. Taiel completed her doctorate in Medicine with board certified in Ophthalmology from Lariboisiere Saint Louis University, Paris, France, in 1993, and her Associate Professor degree in 1998. Dr Taiel completed her internship at academic Paris hospitals, was an Associate Professor of Ophthalmology, served as an Ophthalmology Department Head, and ran Surgical and Medical Ophthalmology private practice. After 13 years of Ophthalmology public and private practice, Dr Taiel has been engaged in the Pharma Industry for 20 years; she brings extensive experience and expertise in drug clinical development, gene therapy, and medical affairs. She started her carrier at Servier company headquarter, and then worked in Ophthalmology area at Pfizer for several years; she then held international and management positions in various therapeutic areas, including both technical and supervision duties, at Eli Lilly Company for many years. Then, as VP Clinical Development, she led Clinical Development and Operations, to develop antisense oligonucleotides in Inherited Retinal diseases at ProQR Therapeutics. She then moved to GenSight-Biologics in 2018, to supervise the Medical Department and lead Gene Therapy programs in Inherited Retinal and Neuro-Ophthalmology diseases, as the CMO of the company. Dr. Taiel has authored numerous protocols and articles published in peer reviewed journals, and made critical contributions to successful clinical development and launch of many products. She brings extensive years of experience from both academic medicine and pharma industry.

Dr. Zeynep Unluturk studied Medicine at the Dokuz Eylul University, Izmir, Turkey and graduated as MD in 2013. She then joined resident Neurology at the Pamukkale University, Denizli, Turkey. She started to work as a clinical neurologist in Giresun University Prof. Dr. A. Ilhan Ozdemir Training Hospital in 2020. After two year public obligator duty in there she started to work University of Health Sciences Kocaeli Derince Training Hospital as a clinical neurologist in 2022.

Dr. Harsha Karur Rajasimha is a healthcare and life sciences scientist, social entrepreneur, and the CEO of Jeeva Clinical Trials. He earned his PhD in Genetics, Bioinformatics and Computational Biology at Virginia Tech (2007). He also founded the Indo US Organization for Rare Diseases, focused on advancing research and advocacy for rare diseases. Dr. Rajasimha received the Sanofi Genzyme’s rare diseases Patient Advocacy Leadership award in 2016. An accomplished scientist, Dr. Rajasimha has over 16 years work experience and 15 peer-reviewed publications, book chapters, and is often invited speaker including keynote addresses at international conferences.

Dr. Cuesta studied Biology at the Autonomous University of Madrid (Spain). In 2003, he joined to Dr. Alvarez-Vallina at the Puerta de Hierro University Hospital, Madrid. After his PhD degree in 2009, in 2011 he moved with Dr Acker-Palmer at the Buchmann Institute for Molecular Life Sciences (Goethe University Frankfurt, Germany). In 2017 he returned to Spain and combined his research in rare diseases with Dr Botella at Centro de Investigaciones Biologicas Margaritas Salas- CSIC with lectures as Associate Professor at the Complutense University of Madrid (UCM). In 2021, he obtained a lecturer position at UCM. 11 out of more than 30 research articles in SCI(E) journals are related with rare disease and drug repurposing.

Roy G. Beran is trained as a consultant neurologist and accredited sleep physician, in addition to working within legal medicine, military medicine and aviation medicine. His qualifications include: MBBS, MD, FRACP, FRACGP, Grad. Dip. Tertiary Ed., Grad. Dip. Further Ed., FAFPHM, FACLM, FRCP, FAAN, FACBS, B Leg. S, MHL and FFFLM (Hon). He is registered with the Australian Health Practitioner Regulation Agency (AHPRA) as a specialist in Neurology, Public Health and Sleep Medicine and was a Designated Medical Examiner for the Civil Aviation Safety Authority, a medical assessor for Dispute Resolution for the State Insurance Regulatory Authority and an assessor for the Workers Compensation Commission of New South Wales (NSW). He is a Conjoint Professor of Medicine at the University of NSW; Professor in the School of Medicine at Griffith University, Queensland; and Professor, Chair, Medical Law, Sechenov Moscow 1 st State University, Moscow, Russia. He was the inaugural Visiting Professor at the International Research Institute of Health Law Sciences at the Southern Medical University, Guangzhou, Guangdong Province, China. He was also a visiting professor to the Macau University and is co-editor of the textbook, ‘Legal Liability in Asia and Australasia’, with Prof Raposo from that university. He is: a founding Fellow of the Australasian College of Legal Medicine; a Past President of the College, having stepped down in 2011, while remaining on Council, and was awarded the second ever Honorary Life Fellowship of the College. In 2019, he was appointed as ‘Co-Head of Faculty’ to convene and co-ordinate the College’s training courses throughout Australia. He is the Australian Governor and was the Secretary General and remains a Vice President of the World Association for Medical Law (WAML), having served on the Organising Committee of numerous World Congresses on Medical Law, presided over the World Congress, in Sydney, in 2004, and successfully bid to host the World Congress, to be held on the Gold Coast, Queensland, in 2022. He was the first Honorary Fellow of the Faculty of Forensic & Legal Medicine of the Royal College of Physicians (London). In Neurology, he is: a Fellow of the Royal Australasian College of Physicians; a Fellow of the Royal College of Physicians, Edinburgh; Corresponding Fellow of the American Academy of Neurologists; and a Member of the Australian and New Zealand Association of Neurologists. He is a Member of the Australasian Sleep Association, serving on various committees thereof. He pioneered the conduct of clinical trials and undertaking research within private practice, having been principal author in publications which included leading academic/tertiary referral institutions. He has published more than 360 papers, book chapters and letters to the editor, presented in excess of 400 papers at national and international meetings and written or edited 17 books, including ‘Legal and Forensic Medicine’, and is on numerous editorial boards, including being the editor in chief of the international journal, Medicine and Law, for the WAML. His research interests include: Concussion; Epilepsy; Legal Medicine; Stroke; Sleep; Neuroepidemiology; and Medical Education. He was an officer in the Royal Australian Navy Reserve (holding the rank of Commander) and was awarded membership of the general division of the Order of Australia, in 2015.

Dr. Priyanka Wadhwa, a medical graduate with an MBA in healthcare administration. She has dedicated more than 12 years of her career to working in the space of rare diseases, breast cancer and women hormone therapies, etc. She works closely with all the Centers of Excellence Centers in India. Currently, she is working on Named Patient programs through which unregistered rare disease medicines can be made accessible for Indian Rare Disease patients.