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Batten disease

This page shows the latest Batten disease news and features for those working in and with pharma, biotech and healthcare.

CHMP backs two rare disease therapies at April meeting

CHMP backs two rare disease therapies at April meeting

BioMarin got a green light for Brineura (cerliponase alfa), an enzyme replacement therapy for the ultra-rare neurodegenerative disease neuronal ceroid lipofuscinosis type 2 (CLN2), a form of Batten disease that ... There is no approved drug to treat the

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