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BioMarin

After the positive data from their CLN2 Enzyme Replacement Therapy Trial, BioMarin are looking at expanded use. We are working with BioMarin and Great Ormond Street to ensure that eligible patients have access to this. If you know any families with children with CLN2 who are not in touch with us please can you ask them to contact us so we can give them information about expanded use when we have this. We aim to keep all families updated on the progress of this.

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