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Potentially Great News For Children With Batten Disease

Posted by Bee For Battens in Research News | 0 comments

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BioMarin Pharmaceutical Inc. announced on the 12th of January interim results from a pivotal study for cerliponase alfa, a recombinant human tripeptidyl peptidase 1 to treat of patients with late infantile CLN2 disease, a form of Batten disease.

The 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.

This interim data represents an important step on a journey to develop a treatment for CLN2 disorder that may be able to slow the course of this fatal disease.

Read the full article here...

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