I've learned to toss a pie in the face of disease

Overall, mortality rates among youth in the United States were higher than 16 comparison countries used in the study.

From baseline to final assessment, all 7 matched cerliponase alfa-treated children under 3 years of age maintained a motor score of 3, representing a "grossly normal gait, signifying a delay in disease onset," stated BioMarin.

These data highlighted the association of moderate air pollution exposure during pregnancy, infancy, and early childhood on pulmonary function at school age.

The third and final episode in our series looks at what is in the pipeline as well as a discussion around FDA guidance.