Prof. Ari Zimran
The Gaucher Unit, Shaare Zedek Medical Center, Jerusalem, Israel
Professor Ari Zimran was the founder and director of the Gaucher Unit at Shaare Zedek Medical Center in Jerusalem from 1990 to 2018, and is currently a senior physician in the Unit. This is the world’s largest referral center for Gaucher Disease (GD), having monitored more than 900 patients to date, and currently treating about 350 patients with enzyme replacement and substrate reduction therapies.
Professor Zimran developed his interest in GD during his fellowship at the Scripps Research Institute, Department of Molecular and Experimental Medicine in La Jolla, California under the mentorship of Professor Ernest Beutler.
Professor Zimran has published more than 320 professional papers and reviews and has edited three books and has been a leader in clinical trials for new treatments for GD, including Cerezyme™, Zavesca™, VPRIV™, Elelyso™, Cerdelga™ and Ambroxol. His most recent research focuses include the relationship between GD and Parkinson, the development of oral enzyme replacement therapy, and gene therapy.
Dr. Aimee Donald
Manchester University NHS Foundation Trust, UK
Aimee Donald is a clinician working in Paediatric Neurology and Inherited Metabolic Disease in Manchester, UK, she is an NIHR Academic Clinical Lecturer at the University of Manchester. Aimee’s interest is in rare neurodegenerative diseases of childhood, and specifically lysosomal storage disorders. Her PhD focussed on the clinical and genetic aspects of neuronopathic Gaucher Disease and sought to better describe the natural history and phenotype of these patients as part of the UK MRC GAUCHERITE study. Her work also explored novel clinical outcome measures in this and other LSDs.
Aimee’s recent experience is in design and delivery of early phase clinical trials of CNS directed therapies in inherited metabolic disease in children. Her interest in the ethical challenges such trials pose led her to undertake a master’s degree in healthcare Ethics and & Law which she hopes to utilise for the benefit of patients in the future, particularly in the design of clinical trials for infants with life-limiting progressive disorders.
