| On behalf of the more than 700 members
of the National MPS Society, we commend today's approval by the US
Food and Drug Administration of Aldurazyme, the first FDA approved
treatment ever developed for individuals with MPS I. Today's approval
marks the culmination of a long effort to create Aldurazyme. Availability
of this treatment will improve care and quality of life for individuals
with MPS I, and provide hope where little existed before.
We will continue to work with the medical community and the general
public to raise awareness of MPS and ML disorders, because early
diagnosis is critical to improving treatment outcomes. ABOUT MPS I
MPS I is a rare, progressive, debilitating disease caused by a deficiency
of the enzyme alpha L-iduronidase that affects an estimated 3,000
to 4,000 people worldwide, including approximately 1,000 in the
United States. Patients who lack this enzyme experience a wide range
of problems related to the disease, including progressive damage
to the heart, lungs, liver, and kidneys and in some cases decreased
mental function. The early signs of MPS I include enlarged head
size, a combination of recurrent ear, nasal, and respiratory infections,
and hernias, which often plague healthy infants. Additional symptoms
may include corneal clouding, coarse facial features, hearing loss,
or joint deformities. ABOUT THE MPS SOCIETY
Founded in 1974, The National MPS Society is the leading organization
dedicated to the needs of patients with MPS and ML disorders. The
National MPS Society's goal is to ultimately find a cure for MPS
and ML disorders, and we hope to achieve this goal by supporting
research, providing support to individuals and their families affected
by an MPS or ML disease, and promoting public and professional awareness. |
