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Children's Hospital Boston to Establish New Treatment Center for Rare Genetic Disorders to Treat More Patients in Years Ahead
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July 16, 2003
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Contact:
Mary-Ellen Shay
617-355-6420
mary.shay@childrens.harvard.edu
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Specialists at Children's Hospital Boston have treated boy since age 4; hospital to establish new treatment center for rare genetic disorders to treat more patients in years ahead.
Eleven-year-old Nicholas Boyce, who was a patient in an experimental study to develop a new treatment for the often deadly genetic disease MPS 1, is now among the first patients in the U.S. to receive the treatment, called Aldurazyme, following the drug's recent FDA approval. Without treatment many MPS1 patients experience a lifetime of pain and a range of other severe health problems. Nicholas has already been very lucky because patients with the most severe form of MPS typically die before age 10.
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Nicholas was first diagnosed with MPS 1 when he was 4 years old, and has been treated by Dr. Allen Crocker, senior associate in medicine at Children's Hospital Boston and a leading expert in childhood metabolic disorders, for nearly a decade. Aldurazyme was developed by Cambridge-based Genzyme Corporation. MPS 1 belongs to the class of rare diseases known as lysosomal storage disorders that also includes Gaucher Disease and Tay Sachs disease. In addition to the development of Aldurazyme, there have been other recent advances in the ability to treat certain LSDs. To bring these new treatment options to more patients from around the world, Children's Hospital Boston has established the new Lysosomal Center for Diagnosis and Treatment, the first treatment center to focus specifically on treatment of these rare and often fatal diseases. The Lysosomal Center will bring together experts from over 10 Children's Hospital Boston departments, including genetics, metabolism, hematology, orthopedics, neurology and cardiology. There are approximately 50 lysosomal disorders that the Center will be equipped to treat.
Nicholas and other patients with MPS 1 lack a certain enzyme that processes naturally occurring carbohydrates in the body. Without this enzyme, carbohydrates accumulate in the body, causing damage to several organs, including lungs, liver and heart, as well as bones, joints and eyes. Without a treatment for MPS 1, Nicholas would be likely to face a lifetime of severe health problems including difficulty breathing, respiratory infections and cardiac problems, skeletal and joint deformities.
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Since Nicholas was diagnosed with MPS 1, his mother Dawn Checrallah of Providence, RI, has been working tirelessly to get her son the treatment that could save his life. As his health deteriorated, she found out about a study for a new treatment called enzyme replacement therapy, and eventually got her son enrolled in the study. Treatment involved an infusion administered in a hospital every week that replaced the missing enzyme in his body. Nick's participation in the national study helped to lead to the development of the first treatment available for MPS 1, which will now be available to thousands of other children living with this terrible genetic disorder.
Nick and Dawn are available for interviews and can discuss their struggle with MPS 1 and their heroic efforts to find a treatment to save Nick's life. Dr. Allen Crocker of Children's Hospital Boston is also available for interviews and can discuss Nick's treatment over the past decade. Dr. Crocker can also help parents to tell if they are at risk for passing MPS 1 or other genetic diseases on to their children, and explain how this new treatment, enzyme replacement therapy, works. Dr. Deborah Marsden, director of the new Lysosomal Center for Diagnosis and Treatment at Children's Hospital Boston is also available for interviews and can outline how the new Center will change the lives of potentially thousands of patients with genetic disorders.
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In addition to arranging for you to speak with the Checrallah family or Drs. Crocker or Marsden about this important milestone in medicine, we can also supply B-roll footage that illustrates the cell culturing and manufacturing process of Aldurazyme. If you are interested, please contact Cara Birrittieri at Children's Hospital Boston, or Gina Cella at Cella Communications.
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Children's Hospital Boston is the nation's premier pediatric medical center. Children's has been ranked #1 among the country's pediatric hospitals in U.S. News and World Report for 13 years in a row. Founded in 1869 as a 20-bed hospital for children, today it is a 300-bed comprehensive center for pediatric and adolescent health care grounded in the values of excellence in patient care and sensitivity to the complex needs and diversity of children and families. Children's Hospital Boston is the primary pediatric teaching affiliate of Harvard Medical School, home to the world's leading pediatric research enterprise, and the largest provider of health care to the children of Massachusetts. For more information about the hospital visit: www.childrenshospital.org.
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