Brock Benroth is a small business owner, and the married father to two daughters: Kate, who had CLN2 and is a Batten angel and her older sister Lauren, who is a carrier. He has joined the board because through his family’s journey with Kate, her participation in a clinical trial, his experience as the Ohio BDSRA chapter president, and their fundraising efforts he has a good connection with other Batten families and the BDSRA that will allow him to put the passion he has for helping people to good use.
Mike Collins works for Numotion as an Assistive Technology Professional (ATP) and Certified Rehabilitation Technology Supplier (CRTS) in Mokena Illinois. Mike has been a member of the board since 2011 and brings his industry knowledge and experience to the board in order to facilitate an exchange of information between Batten families and industry professionals in the challenges we face with Batten Disease. (Read More)
BDSRA is currently accepting applications for the position of Executive Director. Founded in 1987, our long-term vision is a world without Batten disease. Our mission is to support Batten families, fund and facilitate research for treatment and a cure, and advocate for action. Please click here for more information and to apply. (Read More)
From left to right: Dr. Dearborn, Dr. Dang Do, Dr. Dean, Dr. Nelvagal
An Ngoc Dang Do, MD PhD
NICHD, NIH
For many young investigators of rare diseases, having the opportunities to present the research and to discuss in-person ideas for partnerships with other researchers, families and organizations are crucial. This encourages continued commitment and collaboration towards common goals. I experienced this from having attended the 2018 International NCL conference in London and the 2018 BDSRA meeting in Nashville. You have made my attendance at the 2018 NCL conference possible through the BDSRA Emerging Researcher Travel Award. It is a humbling reminder for me that with all the challenges NCL families face daily, you have made a dedication to supporting others. Thank you. Best wishes as we continue to work together towards a better understanding of these diseases.
Shannon Dean, MD PhD
University of Rochester School of Medicine
The opportunity to attend the meeting allowed Dr. (Read More)
We are excited to share this research update from Collaborations Pharmaceuticals. Thank you to all those who have helped to fund the research that has made this a reality. To read more about the partnership between BDSRA and Taylor's Tale please click here.
(Read More)
The BDSRA Board of Directors has several open seats for the term beginning January 2019. If you have expertise you feel would be relevant and beneficial to the organization and are interested in learning more about serving, please contact the BDSRA board at info@bdsra.org by September 10th. Click here for more information. (Read More)
The expansion of Batten Disease treatment options is crucial to our community’s future. We are excited to share this announcement from REGENXBIO who plans to submit an IND for a new gene therapy for CLN2 in 2019. Because of news like this, we continue to be hopeful about progress for patients and families. You can read the full press release here. (Read More)
In April, Shalini Rana, sibling of Shiv Rana CLN6, won third place in the Steger Poetry Prize. The Steger Poetry Prize is a prestigious award presented by celebrated poet Nikki Giovanni at Virginia Tech University. This year there were over 100 entries which were narrowed down to 12 finalists.
Shalini answered a few questions for us about her brother and her prize-winning poem, Scenes//Funeral Day.
Could you tell us a little about you and your brother Shiv? What are some of your favorite memories or special activities you would do with him?
My name is Shalini Rana. I’m from Vienna, Virginia and am a recent graduate from Virginia Tech, where I studied Creative Writing and Professional & Technical Writing. My current work as a poet is informed by experience—namely my experiences involving my family and my brother, Shiv, who suffered from Batten Disease and died before his tenth birthday. I was fourteen at the time. (Read More)
It is an exciting time for Batten research! As new research and potential trials are announced it is important that as a community that we understand the typical path research takes from the lab bench to patient bedside.
Global Genes has developed a wonderful resource illustrating the drug development journey, the Drug Development Roadmap can be found here. Below is an outline of the path a typical treatment must take to approval. Keep in mind that for rare diseases, like Batten disease, the journey can be a little different because of Food and Drug Administration (FDA) programs that can help speed process up.
One such program is the Orphan Drug Act of 1983 which addresses the unique challenges facing rare disease communities by providing incentives to companies that develop treatments for very small patient populations. Other terms you might hear are Accelerated Approval, Priority Review, Fast Track, Breakthrough Therapy, and Pediatric Priority Review Voucher Program. (Read More)
Margie’s trip to Manitoba started with the Batten Connect Information Sessions. The goal of these sessions was to teach the community more about Batten disease and the needs of Batten families. The first session was geared toward teachers, administrators, school personnel, therapists, socials workers, and counselors. Margie presented along with three teachers from the James Nisbet School and Batten moms Tara Gair, Janelle Zazalak and moderator, Bev Maxim. The real-life experiences of the parents present drove home, in very important ways the challenges and wisdom of Batten families. The teachers were able to share with their peers the incredible ways they support their Batten student in school and the way the school community rallies behind the whole family.
Later in the day, there was a session for medical professionals and anyone else interested in learning more about how they can support Batten Families. It is important that medical professionals in all fields understand the progression of the disease so they can better help families prepare for the future and get access to the equipment and support they need. (Read More)
We are excited to share the tentative agenda for the 2018 BDSRA Family Conference with you today. To see this year’s agenda click here: https://bit.ly/2KyZ5D4
As we get closer to July we will post updates on the keynote speakers and describe the sessions in greater detail. Registration is open until June 27th, click here to begin your registration today: https://bdsra.org/bdsra-conference/
If you don’t follow our Facebook conference page be sure to “like” us so that you can get all the conference news and updates: https://www.facebook.com/BDSRAFamilyConference/ (Read More)