Genzyme Convenes 14th Annual North American Lysosomal Storage Disorder Registries Meeting

Genzyme Convenes 14thAnnual North American Lysosomal Storage Disorder Registries Meeting

Thought leaders specializing in Gaucher, Pompe, Fabry and MPS diseases come together to discuss the registries' role in optimizing patient care, now and in the future

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Genzyme, a Sanofi company (EURONEXT: SAN and NYSE: SNY), today announced that the 14th North American Lysosomal Storage Disorder (LSD) Registries Meeting will take place June 13-14 in Chicago, Illinois. Sponsored by Genzyme, this scientific meeting brings together more than 50 registry investigators and 26 speakers, including physicians, academics, and researchers, as well as 100 nurses and genetic counselors to discuss novel insights garnered from studies in observational disease registries.The goal of these studies is to improve clinical care and to shape the future direction of research in the management of patients affected with LSDs. This year's meeting is co-chaired by Pramod Mistry, MD, PhD, Professor of Medicine and Pediatrics and the Director of National Gaucher Disease Treatment Center at Yale University School of Medicine, New Haven, Connecticut and Lorne Clarke, MD, PhD, Professor Medical Genetics, University of British Columbia, Department of Medical Genetics, Vancouver, Canada.

"Over the last two decades, dedicated efforts of registry investigators across the world have uncovered unique insights into LSDs, not possible from single-center studies; these advances have led to improved patient care and informed the design of studies to further advance the field and shape clinical trials," said Dr. Mistry. "This meeting represents a major milestone to assess the current status of observational registries in these rare diseases and to develop a roadmap towards patient-centered outcomes research to further enhance health outcomes for patients."

The theme of this meeting is "Fostering Evidence-Based Medicine through Registries," with special focus on patient-centered outcomes and insights regarding the diagnosis and management of patients affected with LSDs.

Among the topics for discussion:

  • The evolution of registries and best practices in observational research

  • Collecting data on endpoints that are meaningful to the patient

  • Fueling future research via the registries

  • Quality of life issues

  • Emerging outcomes data

  • Enhancing the value of the registry to physicians, payers, and patients

Since 1991, Genzyme has worked closely with the international LSD community to understand disease epidemiology, natural history and long-term treatment outcomes. Having one central repository of data for these rare diseases has been crucial to delineate variable disease progression and develop algorithms to optimize patient management.

"As a registry pioneer, Genzyme has gathered data to advance medical and scientific understanding of rare genetic disorders and to improve the quality of care of patients around the world," said Genzyme's Head of Rare Diseases Rogerio Vivaldi, MD. "As we look forward to the next twenty years, the need to continuously assess the long-term value of therapy will be fundamental to optimize patient outcomes."

The North American LSD Registries are overseen by independent regional boards of scientific advisors. These advisors are external physicians that form the governance body, oversee the scientific integrity and guide research, publications and policy. Genzyme has provided financial support of the operations since 1991.

About Genzyme, a Sanofi Company

Genzyme has pioneered the development and delivery of transformative therapies for patients affected by rare and debilitating diseases for over 30 years. We accomplish our goals through world-class research and with the compassion and commitment of our employees. With a focus on rare diseases and multiple sclerosis, we are dedicated to making a positive impact on the lives of the patients and families we serve. This goal guides and inspires us every day. Genzyme's portfolio of transformative therapies, which are marketed in countries around the world, represents groundbreaking and life-saving advances in medicine. As a Sanofi company, Genzyme benefits from the reach and resources of one of the world's largest pharmaceutical companies, with a shared commitment to improving the lives of patients. Learn more at

About Sanofi

Sanofi, an integrated global healthcare leader, discovers, develops and distributes therapeutic solutions focused on patients' needs. Sanofi has core strengths in the field of healthcare with seven growth platforms: diabetes solutions, human vaccines, innovative drugs, consumer healthcare, emerging markets, animal health and the new Genzyme. Sanofi is listed in Paris (EURONEXT: SAN) and in New York (NYS: SNY) .

Genzyme® is the registered trademark of Genzyme Corporation. All rights reserved.

Sanofi Forward Looking Statements

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts. These statements include projections and estimates and their underlying assumptions, statements regarding plans, objectives, intentions and expectations with respect to future financial results, events, operations, services, product development and potential, and statements regarding future performance. Forward-looking statements are generally identified by the words "expects", "anticipates", "believes", "intends", "estimates", "plans" and similar expressions. Although Sanofi's management believes that the expectations reflected in such forward-looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Sanofi, that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include among other things, the uncertainties inherent in research and development, future clinical data and analysis, including post marketing, decisions by regulatory authorities, such as the FDA or the EMA, regarding whether and when to approve any drug, device or biological application that may be filed for any such product candidates as well as their decisions regarding labelling and other matters that could affect the availability or commercial potential of such product candidates, the absence of guarantee that the product candidates if approved will be commercially successful, the future approval and commercial success of therapeutic alternatives, the Group's ability to benefit from external growth opportunities, trends in exchange rates and prevailing interest rates, the impact of cost containment policies and subsequent changes thereto, the average number of shares outstanding as well as those discussed or identified in the public filings with the SEC and the AMF made by Sanofi, including those listed under "Risk Factors" and "Cautionary Statement Regarding Forward-Looking Statements" in Sanofi's annual report on Form 20-F for the year ended December 31, 2012. Other than as required by applicable law, Sanofi does not undertake any obligation to update or revise any forward-looking information or statements.

Ingrid Mitchell, 617-768-6699

KEYWORDS: United States North America Massachusetts


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