Dear CLN3 families,

As you know we are in regular contact with the groups involved in the Miglustat trial. Today we received this communication from Theranexus:

“We do continue to work on the Phase 3 Study, as you may have seen in a recent press release : Theranexus has been working to optimize the implementation of the pivotal Phase III trial while ensuring FDA and EMA criteria of acceptability, and has decided to focus mainly on the primary efficacy endpoint – visual acuity – which was approved by the agencies, by measuring it solely in a population of young patients in whom the probability of detecting the effects of treatment is much greater. We still explore the various funding options to enable the pivotal trial to begin, this is the critical point.”

We are also in regular contact with Dr Ineka Whiteman who works for the Beyond Batten Disease Foundation. Last week Liz had the opportunity to speak with Mary Beth Kiser, the CEO and President of the Beyond Batten Foundation and shared with her the frustrations felt by families in the UK about the lack of progress with the funding of the trial, to which she expressed her sympathy.

We understand that families will be feeling frustrated, but we hope that this latest update reassures you that the company are still working towards the Phase 3 trial.

We will be arranging a regular meeting for the community in the next couple of weeks so please look out for further details.

Please contact Jo Nightingale if you have any queries, joannanightingale@bdfa-uk.org.uk