Your BDSRA newsletter is here!

September 2017 Volume 28, Issue 6

This month's newsletter is dedicated to all things research. We will be announcing our 2017 Research Grant Awardees, exploring some basic research principles, and sharing quality of life research and tools. Thank you for reading!

2017 Research Grant Awardees

Each year, BDSRA invites proposals from over 200 scientists working in the field to fund the most promising basic and translational (moving to clinical trial) research. Each proposal is peer-reviewed by experts in lysosomal diseases at universities, children's hospitals and in the pharmaceutical industry to provide well-rounded reviews.

We are honored to partner with the following organizations who funded this year's awardees:

BDSRA-Australia, BDSRA-Canada, Drew's Hope, and Noah's Hope/Hope4Bridget.

See the awardees here

Resource Corner

BDSRA is excited to share with you the Child Neurology Foundation's Respite Care Notebook. Over the last year, CNF has worked with patient advocacy organizations, including BDSRA, to build a comprehensive resource for families who are currently using respite care or are thinking about it for the future.

Learn more and download the CNS Care Notebook here

Batten Research Learning Lab

What are Animal Models?

Model systems are an important part of disease research. These model systems can range from simple single cellular systems to larger animal models. The BDSRA has helped to fund many of these animal models to further today's research programs in therapy development.

Read more about animal models here

Therapeutic Strategies in use for Batten disease

There are a variety of therapeutic strategies being used for Batten disease, including, enzyme replacement therapy, stem cell therapy, gene therapy and therapies involving small molecules. In the Batten Learning Lab on our website we talk about each of these therapies and summarize information from scientific publications and other resources to give you an easy to digest update of the latest approaches.

Additional Resources

If you are interested in learning more about specific research areas or scientific concepts we have Ask an Expert Chats on gene therapy basics, the genetics of Batten disease,

functional behavior assessments ,

the importance of biosamples for research

and children epilepsies and cannabinoids .

You can also view all the posters from recent BDSRA Family Conferences on our SlideShare.

If you have any suggestions for Ask and Expert Chat topics email Noreen Murphy at [email protected].


	Project contributor Rebecca Atkinson shares music therapy tools and techniques with children and families at the 2017 family conference.

Quality of Life is

Cornerstone of Initiative

Self-determination, choice, meaningful activities, inclusion, rights, physical and emotional wellbeing. These broad and important ideals drive all we do as a community to make our childrens' lives with Batten disease better, no matter what stage of the disease they are. BDSRA staff and families play an important role in research projects that bring a global understanding of the needs and wants of our kids especially in our schools. Self-determination, choice, meaningful activities, inclusion, rights, physical and emotional wellbeing. These broad and important ideals drive all we do as a community to make our children's lives with Batten disease better, at all ages and stages. BDSRA staff and families are playing an important role in research projects that bring a global understanding of the needs and wants of our kids, especially in our schools.

JNCL and Education by Bengt Elmerskog of STATPED in Trondheim, Norway, et al, is the culmination of a multi-year initiative focused on education and community quality of life. This book, to be published in early 2018, will be used by teachers, parents and caregivers to understand how best to serve those with CLN3 in school and community settings. Over 200 pairs of parents and classroom teachers participated in the first stages of the work along with BDSRA and 20 other organizations. Academic experts have added more from the literature and classroom experts to bring forward tools, trainings and ideas for meaningful classroom experiences.

Click here to see the book outline

BDSRA Announces Board Nominations for

November Elections

The Batten community has had an historic year in treatment and science-with an FDA approved treatment for CLN2, gene therapies moving forward, and new discoveries announced seemingly each week. Help BDSRA keep this momentum as we fulfill our new, expansive strategic plan beginning in 2018. It is an exciting time and we want you to be part of the new vision.
The BDSRA Board of Directors will have three available seats beginning January, 2018. The board and staff seek individuals who are motivated by the opportunities and challenges of fundraising and resource development, governing, research, and outreach to families.

Thank You Edie Dockter!

BDSRA families send a very gracious thank you to Edie Dockter, who has retired after 30 years as the Batten registry coordinator for the Institute for Behavioral Research in Staten Island, NY. With Edie's guidance, the Batten Disease Registry has added more than 1,200 patients since 1987. The registry identifies families with Batten disease and provides a computerized central data bank of clinical information for clinicians and researchers.

Thank you Edie for all that you have done to further Batten disease research.

Changes Announced in the Fall Combined Federal Campaign

For our community members that work in government or the military, you may have been alerted through your Human Resources Director that the significant changes in progress at CFC took longer to implement than anticipated. Therefore, a modified start date of October 2 ^nd has been announced. With this month delay in start date, the campaign will end on January 12 ^th, several weeks longer than in the past. For some, a further delay in start dates can be expected, specifically in the new "zones."