biomarin-pharmaceutical_416x416On the 12th of January BioMarin provided preliminary data from an on-going Phase 1/2 Pivotal Study of BMN 190 for treatment of CLN2 disorder, a Form of Batten Disease. Preliminary results show evidence of disease stabilization.

The pivotal study for BMN 190 (cerliponase alfa), a recombinant human tripeptidyl peptidase 1 to treat patients with late infantile CLN2 disease, a form of Batten Disease. Interim data indicates that in all nine of the BMN 190 patients who have been followed for at least six months and up to 15 months, the treatment appears to show stabilization of the disease compared to the natural history based on a standardized measure of motor and language function.

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