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Shire Enhances its Orphan Drug Pipeline With the Acquisition of a New Clinical Candidate for Metachromatic Leukodystrophy
Date:4/23/2008

>Disease Background

Metachromatic Leukodystrophy (MLD) is in the family of lysosomal storage diseases (LSD's). MLD is an autosomal recessive disease caused by a deficiency of the lysosomal enzyme arylsulfatase A (ASA) which results in an increased concentration of sulphatide in cells of the brains and in non-neural tissue, such as the kidneys and gallbladder. When these sulfated glycolipids accumulate in the brain, they cause a breakdown of myelin, a substance that protects the nerves in the brains and in the rest of the body. This breakdown is what makes MLD a progressive, neurodegenerative disease.

Symptoms and Patient Outlook

Sulfatide accumulation in the central and peripheral nervous system leads to destruction of the myelin sheath (demyelination). MLD has a spectrum of disease which can arise in infants and young children with a range of symptoms, though most are related to motor and cognitive decline. Most MLD patients are normal at birth but often die before age 20, with some patients within the first few years of life. During the final stages all patients reach a decerebrate, vegetative state. The majority of cases are late infantile or juvenile patients. The number of adult patients exhibiting mild forms of MLD is unknown, as adult onset MLD can present with symptoms similar to psychosis.

SHIRE PLC

Shire's strategic goal is to become the leading specialty biopharmaceutical company that focuses on meeting the needs of the specialist physician. Shire focuses its business on attention deficit and hyperactivity disorder (ADHD), human genetic therapies (HGT), gastrointestinal (GI) and renal diseases. The structure is sufficiently flexible to allow Shire to target new therapeutic areas to the extent opportunities arise through acquisitions. Shire's in-licensing, merger and acquisition efforts are focused on products in niche markets with strong intellectual property protection either in the US or Europe. Shire believes that a c
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SOURCE Shire PLC
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