DONATE NOW

? RESEARCH UPDATE - CLN2 DISEASE ?

4 July 2022

Ongoing retinal degeneration in CLN2 disease, despite enzyme replacement therapy.
A study published this week in the British Journal of Ophthalmology shows that retinal degeneration progressed in patients with CLN2, despite intraventricular enzyme replacement therapy with Brineura (cerliponase alfa) which is delivered into the brain ventricles. Degeneration of the retina was particularly pronounced between 56 and 80 months of age.
These findings support the need for retina-directed therapies and the importance of initiation of these therapies as early as possible. A number of clinical studies for retinal-specific treatments are ongoing.
For more information on the study head to: https://pubmed.ncbi.nlm.nih.gov/35772852/