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NEWS Gene Therapy News

Dear all,

We have been working with our international partners, the BDSRA, BDSRA Australia and Baza Retkih Bolesti to dissect recent developments in the batten treatment space.

You will have read the letter from Taysha Gene Therapies published in the last few days (read it here) and although Amicus Therapeutics have not published a community letter as yet, unfortunately they are also pausing gene therapy developments for Batten disease.

We understand this is difficult and extremely frustrating news for many of our Batten families here in the UK and internationally, but we wanted to reassure you that the BDFA and our international partner organisations are committed to working alongside and advocating for all of the Batten families.

We will continue to work together to make a difference.

Following on from this news, the BDFA along with its international partners will be hosting a special virtual session of Ask an expert, as an opportunity for you all to ask any questions you might have regarding the developments.

The panel will include Dr David Pearce, President of Innovation, Research and World Clinic for Sanford Health in Sioux Falls, SD and researcher into Batten disease and Dr Ineka Whiteman, Head of Medical and Scientific Affairs and Head of Research from BDSRA Australia.

The session will be held on Thursday 5th May 2022 at 5pm EDT. (Note- this session will be at 10pm BST in the UK.)

To register for this session please sign up HERE

Please reach out to us at the BDFA if you would like to discuss any of these developments further. Contact Amanda Mortensen at

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