2017 WORLDSymposium Meeting Highlights

Konrad Sandhoff, PhD Received 2017 Award for Innovation and Accomplishment

Each year, WORLDSymposium recognizes one individual for innovation and accomplishment in the field of lysosomal disease research and therapy. This year, the 2017 Award for Innovation and Accomplishment in the field of lysosomal disease research and therapy was presented to Konrad Sandhoff, PhD.
Konrad Sandhoff completed his PhD in biochemistry in Munich. After early research in Munich, Israel and the USA, he became Professor of Biochemistry at the University of Bonn in 1979. Since 2007 he has been a Senior Professor at the LIMES Institute in Bonn, Germany. Professor Sandhoff’s major research interests are focused on molecular life sciences including the analysis and pathobiochemistry of lysosomal (glyco-) sphingolipid storage diseases, the structure and function of lysosomal enzymes and lipid binding proteins, the topology of endocytosis and glycolipid metabolism, and regulation of glycolipid biosynthesis. He has published more than 480 peer-reviewed papers, and has received more than 15 renowned national and international awards. Professor Sandhoff’s early investigation and analysis led to the identification of several biochemically distinct lysosomal diseases, including a variant of GM2-Gangliosidosis which was named “Sandhoff disease“ in 1971.
Professor Sandhoff received the 2017 award at the opening session of WORLDSymposium 2017 on Tuesday, February 14, 2017 at 7:45 AM.
Click here for more information about the Innovation and Accomplishment award.

Christine Lavery, MBE Received 2017 Patient Advocate Leader Award

WORLDSymposium recognized Christine Lavery, MBE, Group Chief Executive for the UK Society for Mucopolysaccharide Diseases, with the 2017 Patient Advocate Leader (PAL) Award.
Christine Lavery founded the United Kingdom (UK) Society for Mucopolysaccharide Diseases (MPS Society) in 1982 following the death of her firstborn son, Simon, from Hunter disease. She started her career in the voluntary sector with ‘Contact a Family’ and for ten years she helped parents establish over 200 rare disease patient organisations. In 1993, she was appointed Chief Executive of the MPS Society, where she pioneered the Patient Advocacy Service. The Patient Advocacy Service is recognised as a gold standard as well a leading organisation that has awarded over £5 million in grants for academic and clinical research.
Christine established the 30 year old MPS International Network as well as serving on four medical advisory panels including the National Health Service England Lysosomal Storage Disorders Advisory Board.
In 2002, Christine was awarded the Member of the British Empire (MBE) for her services to metabolic diseases by Her Majesty (HM) Queen Elizabeth II in the New Year’s Honours List. In 2006 she received ‘A Life Time Award’ from the International MPS Community at the 2006 International Symposium on Mucopolysaccharide and Related Diseases.
In 2013 Christine launched MPS Commercial, a wholly-owned subsidiary of the MPS Society, to enhance logistical support to patients with rare diseases and their families participating in commercial and academic clinical trials and health technology assessments.
WORLDSymposium honored Christine with the 2017 Patient Advocate Leader Award on Thursday, February 16, 2017 at 6:00 PM at the WORLDSymposium Banquet and Award Ceremony.
Click here for more information about the Patient Advocate Leader award.

Elsa Shapiro, PhD, Delivered Keynote on Wednesday, February 15, 2017

Elsa Shapiro, PhD
Elsa Shapiro, PhD, is Professor of Pediatrics and Neurology in the Division of Pediatric Behavioral Neuroscience at the University of Minnesota. She is retired, but remains a part-time faculty member. She has a PhD in Psychology from the University of Minnesota, completed her internship and post-doctoral training at National Children’s Medical Center in Washington, DC, and is board certified  in clinical psychology. Dr. Shapiro is known for research in neurobehavioral and neuroimaging manifestations of genetic neurodegenerative disorders. She developed methods of longitudinal assessment of neurocognitive functions, delineated the neurocognitive phenotypes of several genetic disorders, studied the relationships between quantitative neuroimaging and neuropsychology in treated and untreated children, and examined the characteristics of dementia in children with neurodegenerative disease. She has authored more than 100 peer-reviewed publications and invited chapters. Recently, she was the co-Principal Investigator of the NIH-supported Lysosomal Disease Network, and Principal Investigator of Longitudinal Studies of Brain Structure and Function in the Mucopolysaccharidoses until 2014. She was also co-Principal Investigator of a Natural History study of MPS III. She currently offers consulting services to provide assistance with neurocognitive endpoints in patients with rare diseases.
Dr. Shapiro also has a strong interest in the effects of poverty on the developing brain, and previously led a large study of the effects of lead burden and other social and biological variables on the cognitive development of high-risk inner city children. She has also consulted on the effects of cerebral malaria and HIV on neurodevelopment in Ugandan children. In addition to her research, she was the founder of the Pediatric Neuropsychology clinic and training program at the University of Minnesota in 1974, training many post-doctoral fellows. She has mentored 11 fellows and junior faculty in research in neurodegenerative diseases. She now lives in Portland, Oregon.
Dr. Shapiro delivered a Keynote Address at WORLDSymposium 2017, Understanding and Measuring Neurodegeneration in Childhood Onset Lysosomal Diseases, on Wednesday, February 15, 2017 at 7:45 AM.

Keynote Address on Thursday, February 16, 2017 presented by Richard Moscicki, MD, Deputy Center Director for Science Operations, U.S. FDA

Richard Moscicki, MD
Richard Moscicki, MD, from the U.S. Food and Drug Administration (FDA) presented the Thursday, February 16th Keynote Address at WORLDSymposium 2017.
Richard (Rich) A. Moscicki (Mo-shis-ke), MD, joined the U.S. Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER), as Deputy Center Director for Science Operations. A nationally recognized expert in clinical research and development, Dr. Moscicki brings to the position executive direction of Center operations and leadership in overseeing the development, implementation, and direction of CDER’s programs.
Before joining CDER, Dr. Moscicki served as senior vice president (SVP), Head of Clinical Development at Genzyme Corporation. He joined Genzyme in 1992 as medical director and became the chief medical officer and SVP of biomedical and regulatory affairs in 1996 — holding that post until 2011. Over the past two decades, Dr. Moscicki has been responsible for worldwide global regulatory and pharmacovigilance matters, as well as all aspects of clinical research and medical affairs for the company.
Dr. Moscicki received his medical degree from Northwestern University Medical School. He is board certified in internal medicine, diagnostic and laboratory immunology, and allergy and immunology. He completed his residency with a focus on immunology, followed by a four-year fellowship at Massachusetts General Hospital (MGH) in immunology and immunopathology. He remains on staff at MGH and on the faculty of Harvard Medical School.
Dr. Moscicki presented An FDA Perspective on Rare Disease Drug Development at WORLDSymposium 2017 on Thursday, February 16, 2017, from 7:45-8:15 AM.

New Treatment Award

Each year WORLDSymposium recognizes important achievements in therapy for lysosomal diseases. In 2017 one new treatment was recognized for achieving regulatory approval. The WORLDSymposium 2017 New Treatment Award went to migalastat (Galafold™) which provided clinical data meriting approval by the European Medicines Agency and European Commission in 2016.


Congratulations to the ten individuals selected to receive the WORLDSymposium Young Investigator Award for 2017. The award is a partial scholarship towards attendance at WORLDSymposium 2017. Numerous individuals submitted an application for the award, and the review process was difficult due to the excellent caliber of all the applicants. WORLDSymposium would like to congratulate all of the applicants for their hard work. The following individuals received the WORLDSymposium Young Investigator Award for 2017 and were recognized at the closing banquet and award ceremony on Thursday, February 16, 2017:

  • Brendan Beaton
  • Carolina Cardona
  • Shahzeb Hassan
  • Shih-hsin Kan
  • Francyne Kubaski
  • Reid Martin
  • Stephanie Newman
  • Li Ou
  • Vincent Puy
  • Dora Lucia Vallejo Ardila

Click here for more information about the Young Investigator Awards.