12 presentations and three satellite symposia showcase Shire’s commitment to improving understanding and treatment of these life-altering disorders
Cambridge, Ma. – February 5, 2018 – Shire plc (LSE: SHP, NASDAQ: SHPG), the global biotechnology leader in rare diseases, today announced that it will have 12 presentations, including 11 posters and one oral presentation, at the 14th annual WORLDSymposium™ 2018 in San Diego, Calif., February 5-9. Shire will share research data for four lysosomal disease areas, including Hunter syndrome (also known as Mucopolysaccharidosis type II or MPS II), type 1 Gaucher disease, Fabry disease and metachromatic leukodystrophy (also known as MLD).
The company will also sponsor three satellite symposia and host an exhibit at WORLDSymposium™ (booth #301).
“We are proud to participate again at this year’s WORLDSymposium, a unique conference that focuses exclusively on lysosomal diseases, an area that formed the beginning of Shire’s strong commitment and focus on rare diseases,” said Hartmann Wellhoefer, M.D., Vice President, Head of Rare Diseases and Internal Medicine, Global Medical Affairs, Shire. “This meeting brings together the world’s leading experts in the rare disease community to exchange the latest scientific knowledge and data about lysosomal diseases, along with advocacy groups who share valuable patient insights and perspectives. We look forward to sharing our data and contributing to the dialogue regarding earlier diagnosis, treatment and management of rare diseases.”
The congress is focused on sharing the latest information for lysosomal diseases, a collection of some 50 clinical syndromes, each resulting from the deficiency of a particular protein and causing a significant amount of disability and disease burden1.
Shire’s presence at the meeting includes the following key presentations, which are intended for scientific discussion only:
Shire will also be sponsoring several symposia at the meeting:
Sponsored by Shire Commercial
Sponsored by Shire Medical
About the Genetic Diseases Franchise
Shire is a dedicated champion for people with rare diseases, which are typically inherited illnesses. Shire’s Genetic Diseases franchise has a strong legacy in developing therapies for lysosomal diseases, with a portfolio that includes commercial products and a robust R&D program with late-stage investigational therapies and pipeline candidates. The company’s commercial products address the following three disease areas:
Non-US HCPs: For additional information about agalsidase alfa, click here.
For additional information about idursulfase, click here.
For additional information about velaglucerase alfa, click here.
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Shire is the global leader in serving patients with rare diseases. We strive to develop best-in-class therapies across a core of rare disease areas including hematology, immunology, genetic diseases, neuroscience, and internal medicine with growing therapeutic areas in ophthalmics and oncology. Our diversified capabilities enable us to reach patients in more than 100 countries who are struggling to live their lives to the fullest.
We feel a strong sense of urgency to address unmet medical needs and work tirelessly to improve people’s lives with medicines that have a meaningful impact on patients and all who support them on their journey.
1 Fuller M, Meike PJ, Hopwood JJ. Epidemiology of lysosomal storage diseases: an overview. In: Mehta A, Beck M, Sunder-Plassmann G, editors. Fabry Disease: Perspectives from 5 Years of FOS. Oxford: Oxford PharmaGenesis; 2006. Chapter 2.
2 Fabry Disease. National Organization for Rare Disorders. Accessed January 2018. Available at: https://rarediseases.org/rare-diseases/fabry-disease/
3 Meikle PJ et al. Prevalence of Lysosomal Storage Disorders. JAMA. 1999. 281(3):249-54.
4 Wraith JE et al. Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and (3):267-77.
5 Wraith JE. Idursulfase for enzyme-replacement therapy in mucopolysaccharidosis II. Therapy. 2007. 4(3):231-240.
6 Muenzer J et al. A phase I/II study of intrathecal idursulfase IT in children with severe mucopolysaccharidosis II. Genet Med. 2016. 18(1):73-81.
7 Guggenbuhl P, Grosbois B, Chalès G. Gaucher disease. Joint Bone Spine. 2008; 75(2):116-124.
8 Grabowski GA. Phenotype, diagnosis, and treatment of Gaucher’s disease. Lancet. 2008; 372:1263-71.