EURORDIS is conducting an internet-based survey in an effort to better understand how well patients are engaged in medical care, including the relationship with health care providers, the understanding of the therapy (its efficacy, possible difficulties, benefits as they see…
The first meeting of the BATCure group took place at UCL in London on January 27-29. It was a very exciting to finally be starting on the project with over 30 scientists, research students and clinicians attending from Denmark, Germany,…
Join Royal College of Pediatrics and Child Health for the BPSU's 30th anniversary Rare Diseases Conference, taking place on 23rd February 2016 at the University of Birmingham. The conference will bring together paediatricians, trainees and other healthcare workers with an…
UCL School of Pharmacy is carrying out a study to investigate the views of parents and carers about administering medicines to children via the rectal route. The survey will ask some demographic and background information before presenting three scenarios, where…
Congratulations to Sophia who has been awarded the degree of PhD today. This a fantastic achievement and one we can all take great pride in as all your efforts funded her studies. This is an important career step for Sophia and rewards…
Breaking news for Batten Disease "Our study opens up the possibility of a one-and-done treatment for this form of Batten disease," said Beverly Davidson, Ph.D., director of the Raymond G. Perelman Center for Cellular and Molecular Therapeutics at Children's Hospital…
This October our Family Conference was held at the Village Hotel in Coventry. Families, staff and professionals started arriving on the Friday, meeting up with old friends and making new ones. The AGM on Saturday morning, included a keynote speech…
Threats to science funding is a threat to us all, please visit and send an e-postcard to the Chancellor about why science and research is vital for Batten disease. The government is once again threatening research with cuts of 25%…
The lovely Harrison family whose son James has CLN1 (Infantile Batten disease) will be raising awareness on This Morning on Monday 5th October. Please watch and let people know, a great platform to raise awareness.
Birmingham Children's Hospital is launching a £3.65 million appeal to raise money to build the UK's first rare diseases centre for children. Read more here