2016 WORLDSymposium Meeting Highlights

Christopher P. Austin, MD, Delivered Keynote at WORLDSymposium 2016

Dr. CHRISTOPHER P. AUSTIN, MDChristopher P. Austin, MD, delivered the Keynote Address at WORLDSymposium 2016. Dr. Austin is director of the National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health (NIH). Austin leads the Center’s work to improve the translation of observations in the laboratory, clinic and community into interventions that reach and benefit patients — from diagnostics and therapeutics to medical procedures and behavioral changes. Under his direction, NCATS researchers and collaborators are developing new technologies, resources and collaborative research models; demonstrating their usefulness; and disseminating the data, analysis and methodologies for use by the worldwide research community.

Dr. Austin delivered his address, Catalyzing Translational Innovation, at WORLDSymposium 2016.

Barbara Wedehase Received New 2016 Patient Advocate Leader (PAL) Award

Barbara WedehaseWORLDSymposium and Lysosomal Disease Network gave thanks to someone very special who has contributed directly to lives of countless individuals dealing personally with a lysosomal disease. With a new award for Patient Advocate Leaders, the Network recognized Barbara Wedehase. Barbara Wedehase has been the Executive Director of the National MPS Society for 15 years and has overseen the tremendous growth of the Society and its programs, including over $6.5 million in research funds awarded. She has a Masters in Social Work from Washington University in St. Louis, Missouri and is a Board Certified Genetic Counselor. She was a Clinical Assistant Professor of Pediatrics in the Division of Genetics and Metabolism at the University of North Carolina prior to her work with the National MPS Society. On a national level, Ms. Wedehase served on the Steering and Executive Committees of the Lysosomal Disease Network and has been the energizing, founding leader of the Lysosomal Disease Network’s Council of Patient Advocacy (COPA) groups. She participated in an expert working group for a lysosomal disease educational initiative; served on the MPS I Technical Expert Panel for admission of MPS I to the newborn screening panel; and helped to establish the LSD Research Consortium and the awareness program, Join the Search for Patients with MPS. Internationally, Ms. Wedehase served on the Program Committees of the International MPS Societies Network and the International MPS Symposiums; was a member of the Ethics Advisory Group for the MPS VI gene therapy clinical trial.

Dr. Emil Kakkis Received 2016 Award for Innovation and Accomplishment

Emil Kakkis, MD, PhDEach year, WORLDSymposium recognizes one individual for innovation and accomplishment in the field of lysosomal disease research and therapy. This year, the 2016 Award for Innovation and Accomplishment in the field of lysosomal disease research and therapy was presented to Emil Kakkis, MD, PhD. Over the last 22 years Dr. Kakkis is best known for his work developing novel treatments for rare diseases and policy issues affecting rare disease treatment development. Dr. Kakkis began his career as Assistant Professor at UCLA where he initiated research on enzyme replacement therapy for mucopolysaccharidosis type I. More recently, he founded the non‐profit EveryLife Foundation for Rare Diseases, dedicated to the acceleration of biotech innovation for rare diseases through practical and scientifically sound improvements to development strategies, regulatory policy and laws. Dr. Kakkis is currently President and Chief Executive Officer of Ultragenyx Pharmaceutical where he leads a drug development team working on multiple rare and ultra‐rare disease treatments.

New Treatment Award

Each year WORLDSymposium recognizes important achievements in therapy for lysosomal diseases. In 2016 one new treatment was recognized for achieving regulatory approval. The New Treatment Award went to sebelipase alfa (Kanuma™) which provided clinical data meriting approval by the European Medicines Agency and by the U.S. Food and Drug Administration.

WORLDSymposium 2016 Young Investigator Awards

Eight individuals received the WORLDSymposium Young Investigator Award for 2016. The award was a partial scholarship towards attendance at WORLDSymposium 2016. Numerous individuals submitted an application for the award, and the review process was difficult due to the excellent caliber of all the applicants. We would like to offer our appreciation to all of the applicants for their hard work. The following individuals received the WORLDSymposium Young Investigator Award for 2016:

  • Elma Aflaki
  • Allison Bradbury
  • Fu-Pang Chang
  • Camila de Britto Para de Aragao
  • Chrissa Dwyer
  • Arunabha Ghosh
  • Zhirui Jiang
  • Mari Mori

Nih-Funded Lysosomal Disease Network Fellows

The following information is being provided as a courtesy to the Lysosomal Disease Network (LDN), in recognition of the LDN’s important contributions to lysosomal disease research:

Each year of the LDN’s NIH funding cycle, two postdoctoral Lysosomal Disease Network Fellows are selected, based upon their submitted applications. One LDN Fellowship is funded through LDN NIH funds. The University of Minnesota Medical School provides $50,000 matching funds for an additional postdoctoral Fellow, to be located at the University of Minnesota in the Twin Cities. Fellows are selected by the LDN Steering Committee, and can elect training in any clinically-related field such as clinical genetics, neurology, neuropsychology, or any field of medicine that might provide research or clinical service for patients with lysosomal disease. They are required to present their research at a major medical conference of their choosing, and at the recurring Conference on Clinical Research for Rare Diseases (CCRRD) presented by the Rare Diseases Clinical Research Network.

  • Zahra Karimian (LDN and Genzyme-Sanofi Fellow)
  • Li Ou (LDN Fellow)
  • Melani Solomon (LDN Fellow)