Many of the CLN2 families in England have been involved in the trials or on the compassionate use scheme for the new enzyme replacement treatment called Brineura. On Friday 22 Feb 2019, NICE did not recommend the treatment.
We are have launched a crowdfunder to raise funds to enable us to campaign to reverse this decision. As you will understand, as a small charity, everything we do needs to be funded, especially when they are so important. So please make a donation and send this link to your friends, families, colleagues and contacts: https://www.crowdjustice.com/case/brineura-denied/
We will update these pages with actions you can take to support our campaign.
Thank you for your support.