Shire Human Genetic Therapies Inc.

Shire Human Genetic Therapies Inc., operating as Takeda, focuses its clinical research on late metachromatic leukodystrophy (MLD), a rare inherited lysosomal storage disorder affecting the nervous system. The studies center on children with late infantile MLD and address the progression of motor impairment associated with the disease.

• Gross motor function decline
• Locomotion preservation
• Neurologic disease progression

The sponsor’s research activity in this area reflects an emphasis on rare neurodegenerative disease and the clinical consequences of impaired motor control in affected children.