Genzyme in the UK

Company Info

Genzyme Supports International Pompe Day 2014

Date: April 15, 2014

Oxford, UK. – Genzyme, a Sanofi company, today announces its support of International Pompe Day, the first time the annual event is being held, to highlight awareness of this rare neuromuscular disease. The inaugural International Pompe Day 2014 is being marked with the theme “Together We Are Stronger”.

Pompe disease is a rare and debilitating muscle disease affecting both children and adults. The infantile-onset form of the disease generally manifests within a few months of birth while the late-onset form appears anytime during childhood or adulthood in a more gradual fashion. Both types are generally characterised by progressive muscle weakness and breathing difficulty, but the severity of the disease can vary widely depending on the age of onset and the extent of organ involvement.

Brendan Martin, General Manager at Genzyme UK and Ireland, said: “Genzyme has a long history of involvement with Pompe and supporting patients and families affected by this debilitating disease. We are pleased that the Pompe community is coming together to raise international understanding and awareness of this rare disease. Genzyme has pioneered the development and delivery of transformative therapies for patients affected by rare and debilitating diseases for over 30 years, and we continue to pursue collaborations and research to try to find additional treatments for these diseases.”

Researchers estimate that Pompe disease occurs in 1 in 40,000 live births.1, 2 At present, Pompe disease is thought to affect all races. Some ethnic groups may experience Pompe disease at higher rates, however. In the African-American population, the incidence of the infantile-onset form has been estimated to be much higher at approximately 1 in 14,000.3

Newborn screening for Pompe disease is currently performed in very few countries around the world, most notably in Taiwan. However, the U.S. Discretionary Advisory Committee on Heritable Disorders in Newborns and Children (DACHDNC) has recently recommended that Pompe disease be added to a list of diseases for which newborns in the U.S. should be screened.

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. That 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.

About Genzyme in the UK

Genzyme is a significant force in the UK biotechnology sector focused on improving the lives of patients with rare genetic diseases and multiple sclerosis. First established in the UK in 1981, Genzyme employs 450 people at two sites. Genzyme Therapeutics in Oxford is the company’s UK commercial base marketing products for the treatment of patients with chronic debilitating diseases. These include genetic diseases such as lysosomal storage disorders (LSDs) – characterised by a lack of enzymes essential to healthy biological processes – and multiple sclerosis – a complex neurological disease affecting the central nervous system. Our portfolio also includes a therapy which aids in the management of thyroid cancer.

Genzyme’s manufacturing facility in Haverhill, Suffolk, now integrated with Sanofi’s Pharma Operations Unit, has undergone a dramatic building programme since its purchase in 1982 and now employs 380 people at a site with two large-scale manufacturing plants, a pilot plant, a development centre, warehouse and other associated facilities. The Haverhill facility manufactures the active ingredients in therapies for patients undergoing renal dialysis and is a major global distribution centre for Genzyme’s products for genetic diseases. Learn more at

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1. Ausems MG, Verbiest J, Hermans MP, et al. Frequency of glycogen storage disease type II in The Netherlands: implications for diagnosis and genetic counseling. Eur J Hum Genet 1999 Sep; 7(6): 713-6.

2. Martiniuk F, Chen A, Mack A, et al. Carrier frequency for glycogen storage disease type II in New York and estimates of affected individuals born with the disease. Am J Med Genet 1998; 79: 69

3. Hirschhorn, Rochelle and Arnold J. J. Reuser. Glycogen Storage Disease Type II: Acid Alpha-Glucosidase (Acid Maltase) Deficiency. In: Wonsiewicz M, Noujaim S, Boyle P, eds. The Metabolic and Molecular Bases of Inherited Disease. 8th Edition. New York: McGraw-Hill; 2001; 3389-3420.


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Contact Info:

Genzyme Therapeutics Ltd
4620 Kingsgate
Cascade Way
Oxford Business Park South
Tel: +01865 405200
Fax: +01865 774172

Contact Info:

Genzyme Ltd, Haverhill Operations
12 Rookwood Way
Tel: +01440 703522
Fax: +01440 716269