General: 07354 486 586   |   Fundraising: 07511 821 181   |   Support: 0800 046 9832  
Biomarin Logo

BioMarin would like to announce that Great  Ormond Street Hospital (GOSH) in London will be joining the European clinical study centres participating in the BioMarin BMN190 trial in the immediate future.  At the time when GOSH initiates the study, the Evelina Hospital will close as a study centre, and families will transition to GOSH for their treatment.  BioMarin believes this change is in the best interest of patients and will provide the needed support and expertise.  It is our desire to maximize every opportunity available to ensure the most accurate and efficient outcome of the trial for all those affected by the disease.

Dr Williams and her team at Evelina Hospital are assisting with the smooth transition of all aspects of the BMN 190 study for late infantile CLN2 patients who are being treated at Evelina, as well as those currently under consideration for eligibility at this centre.

BioMarin remains deeply committed to this clinical program, and to the patients and families.

Questions regarding the above change in study conduct in the UK should be forwarded to Ms. Andrea West, Chief Executive at the BDFA (01252 416110, andreawest@bdfa-uk.org.uk)  who is in regular contact with BioMarin.

Donate to the BDFA

With your support we can help support families living with the devastating diagnosis of Batten disease

Thank you for making a donation to the work of the BDFA.

The BDFA receives NO funding from Government and are only able to carry out our work because of the strong commitment of our volunteers and fundraisers.

Thank you so much for your support

How your donation can help

£5 a month
provides a support and information
folder for a newly diagnosed family

£20 a month

helps to run our family support services

£50 a month
enables us to run training workshops
for professionals to educate them on
Batten disease