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Genetic Disease

Genzyme is widely recognized as a global leader in research, product development, and outreach to the medical and patient communities for rare genetic diseases known as lysosomal storage disorders (LSDs). In 1991, Genzyme introduced the first product ever approved to treat a lysosomal storage disorder, Ceredase® (alglucerase injection). This first-generation enzyme replacement therapy product brought new hope to patients suffering from Type I Gaucher disease. Since then, Genzyme has developed a second-generation Gaucher disease product, Cerezyme® (imiglucerase for injection), and introduced Fabrazyme® (agalsidase beta) for Fabry disease, Aldurazyme® (laronidase) for Mucopolysaccharidosis I (MPS I) and Myozyme® (alglucosidase alfa) for Pompe disease. As part of its commitment to new approaches to LSDs, Genzyme is developing a treatment for ASM-deficient Niemann Pick disease, a degenerative and fatal LSD.

In addition to enzyme replacement therapy, other technology platforms such as cell and gene-based therapies are also being evaluated for use in these diseases. In particular, Genzyme is exploring the potential utility of gene therapy to correct the central nervous system abnormalities associated with several LSDs.
Genetic Disease publications
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Renal Disease

Genzyme is enhancing the treatment of chronic kidney disease with its phosphate binder, Renagel® (sevelamer hydrochloride). Nearly all patients on hemodialysis take a phosphate binder which, before Renagel was introduced, was typically aluminum or calcium-based. Renagel is the only calcium-free, metal-free non-absorbed phosphate binder on the market. Genzyme has expanded its renal business with Hectorol, a line of vitamin D2 pro-hormone products used to treat secondary hyperparathyroidism in patients on dialysis and those with earlier stage chronic kidney disease.
Renal Disease publications
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Oncology

Genzyme Oncology is building a foundation in cancer treatment with a strong focus on antibody and small molecule therapies. Genzyme currently has two marketed leukemia products, Campath® (alemtuzumab for injection) and Clolar® (clofarabine) for intravenous infusion. The company is also focused on new treatments for cancer patients through both internal research and external collaboration. In conjunction with our partners, Genzyme is working to develop therapeutics that target the regulation of cellular level processes, such as antiangiogenesis, cell death, proliferation, and metastasis.
Oncology publications
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Transplant/Immune Disease

The field of transplantation medicine has evolved rapidly, particularly where management of acute organ rejection is concerned. While in the past, organ loss often occurred one to two years after transplantation, the introduction of drugs such as Genzyme’s Thymoglobulin® (anti-thymocyte globulin, rabbit) has significantly improved the success rate of these surgeries. In addition to treating kidney transplant rejection, Thymoglobulin is currently being explored in living donor kidney, bone marrow and liver transplantation. Genzyme is also working internally, and with partners, to develop a variety of new protein and small molecule approaches to a diversity of autoimmune and other diseases.
Transplant publications
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Immune Disease publications
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Orthopaedics

Genzyme is a global leader in the field of orthopaedics, with products on the market and a promising group of products in the development. The leading product in this area is Synvisc® (hylan G-F 20), a viscosupplementation treatment for relieving knee pain associated with osteoarthritis. Genzyme also markets Carticel® (autologous cultured chondrocytes), a cell therapy that repairs damaged knee cartilage, and is pursuing development of next generation cartilage repair products through the combination of biomaterials and biological therapeutics.
Orthopaedics publications
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Adhesion Prevention

Genzyme has developed a suite of biomaterials used to help improve the outcome of certain types of surgeries. Its Sepra line of hyaluronic acid-based products has been clinically shown to reduce the incidence of adhesions following general abdominal and gynecologic surgical procedures. With world-class research and development capabilities, and nearly two decades of experience working with biomaterials, Genzyme is expanding its presence in adhesion prevention through the development of laparascopically applied formulations.
Adhesion Prevention publications
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Cardiovascular Disease

Genzyme is a pioneer in exploring both gene therapies and cellular therapies as potential treatment modalities for serious cardiovascular diseases. With clinical programs employing both therapeutic methods, Genzyme is focused on treating ischemic diseases characterized by inadequate blood flow and poor cardiac function. In order to foster clinical development and preclinical research in cardiac cell therapies, Genzyme and Medtronic Corporation have formed a joint venture, MG Biotherapeutics.
Cardiovascular Disease publications
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Diagnostic Products and Services

Genzyme Diagnostics, through its partners and distributors, offers a novel line of products for the diagnostics industry and the clinical laboratory. Genzyme continues to develop new tests for diagnosing a variety of indications including heart disease, diabetes, pancreatitis, infectious disease, emergency medicine and women’s health.

Genzyme Genetics is a leading provider of reproductive and oncology diagnostic testing services. In the area of reproductive testing, Genzyme focuses on technology that will allow information to be provided on a range of diseases from a single patient sample. In the oncology testing arena, Genzyme’s focus is on creating new tests that aid in the diagnosis, prognosis, and monitoring of patients with cancer. In addition to internal research, Genzyme Genetics has a long-standing history and commitment to partnerships with academic and commercial laboratories.

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On this page:
Genetic Diseases
Renal Disease
Oncology
Transplant/Immune Diseases
Orthopaedics
Adhesion Prevention
Cardiovascular Disease
Diagnostic Products and Services

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