We are pleased to say that a further meeting of the NICE Highly Specialised Technology (HST) Committee was held in Manchester yesterday about the Brineura treatment for CLN2. The meeting was attended by two patient experts who are both mothers…
Registration for NCL 2018 is now open on the conference website: http://www.ncllondon2018.com Please contact conference organisers Bioscientifica who can provide details of discounted rates for families: ncl2018@bioscientifica.com
NICE say NO to the use of Cerliponase Alfa for children with CLN2 (Late Infantile Batten disease) BDFA Summary of NICE Decision NICE ECD Report SIGN THE PETITION HERE This decision means that children diagnosed with this devastating disease in…
Click here to download the Paper This paper addresses some findings and experiences from the Juvenile Neuronal Ceroid Lipofuscinosis and Education Project (2014 - 2017). Juvenile Neuronal Ceroid Lipofuscinosis (JNCL) is also called CLN3 disease, Spielmeyer-Vogt Syndrome and Batten Disease.…
Help us produce the insurance information you need! Accessing appropriate insurance can be a significant problem for many patients and families affected by genetic conditions. Patients and families affected by genetic conditions often find it difficult or impossible to access…
Despite the hopeful news of a European licence for Brineura - the new and only treatment for children diagnosed with CLN2 (Late Infantile Batten disease) - NHS England has now denied access to this only hope for children and families…
Please join us on Thursday 8th June (11.00 – 16.00) at the RVC, London, to find out more about the work of Dr Russell’s laboratory, including their work as part of the BATCure research project. Dr Russell has great expertise…
The BDFA is delighted to announce that in the last week we have received very positive news from the European Medicines Agency (EMA) and the U.S Food and Drug Administration (FDA) on the approval of Brineura as the first treatment…
Professor Tristan McKay and the BDFA would like to invite parents and carers to a Laboratory Open Day on Thursday 30th March 2017 at MMU. The day will run from 10.30am-3.30pm with a light lunch provided. BATCure is a 3-year research project with…
Bears4Rare is an initiative from Shire which aims to bring public attention to the difficulty rare disease patients face accessing treatments. Bears4Rare aims to unite the patient voice to highlight the universal challenges faced by people in the UK living…