Affected Families: We need your help!

Dear Families,
Over the last 9 years, Beyond Batten Disease Foundation (BBDF) has funded research for a treatment or cure for juvenile Batten (CLN3) disease. That research has led to a medical discovery: a treatment that slows the progression of the disease in Batten models.
We are now laser-focused on gaining approval from the US FDA to conduct a clinical trial to treat children with CLN3 disease. If successful, the regulatory pathway we have chosen will lead to approval of the new medicine for all patients, wide distribution, make the drug eligible for insurance coverage, and open the door for treatment of other forms of Batten and lysosomal storage diseases.
We need your help! Anecdotal information from the patient community about their experiences can be very helpful in supporting our case if included as part of our submission package to the FDA. Please take 2 minutes to fill out this short survey to provide important information about your personal experiences, if any, with Trehalose and/or Miglustat.
The survey is confidential and will be anonymous unless you choose to share your contact information. Participating in the survey will in no way affect eligibility for this or any other trial.
Please let us know if you have questions or would like additional information.
With gratitude for all you do for your own family and the broader Batten community. We are grateful for your partnership in this effort.
Look forward to hearing from you!
Mary Beth Kiser
President and CEO


Giving Tuesday Thanks


We are so grateful to all the Batten families and supporters who created fundraisers on our Facebook page or made donations on #GivingTuesday! We made close to $15,000 and anticipate receiving matching of up to $1000 for each fundraiser thanks to Facebook and the Bill & Melinda Gates Foundation! Facebook fundraisers are an easy way to support BBDF’s research efforts and to help fund the treatment that is now in sight for juvenile Batten disease! You can create a fundraiser at any time and for any reason — maybe in honor of your birthday or another holiday, or simply just because! Just go to our Facebook page and click on “fundraisers” on the left side of the page. It will walk you through the steps. It’s very easy! Thanks again to all who made this #GivingTuesday so successful for Beyond Batten Disease Foundation!

Inspiring Family Takes on Philadephia Marathon

Seven-year-old Garrett was diagnosed with Batten disease at the age of four. In dealing with his blindness and inability to walk independently, his family found the importance of wellness of both body and mind. They have also come into contact with many other families who face similar challenges and would love to exercise and enjoy the outdoors with their loved ones. Through this, Garrett the Grand Wellness has evolved. Read the full press release on their attendance at the marathon here.

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Sharon Terry at TedMed 2016

Sharon Terry is a mother of 2 children who have a rare disease known as pseudoxanthoma elasticum (PXE). Watch her inspirational speech where she discusses how she and her husband worked to establish mandates that require researchers to share biological samples and work together. Beyond Batten Disease Foundation has followed that model since its inception.


ucm475291The U.S. Food and Drug Administration’s (FDA) patient representative program has existed since 1999 and is integral to fulfilling FDA’s strong commitment to ensure that the needs and choices of patients – as well as their families, caregivers, and advocates – are incorporated in ever greater ways in the work they do.

Recently, the FDA met with 21 inspirational patients and patient caregivers who have made the extraordinary commitment to become FDA patient representatives. These volunteers were in Washington to participate in a two-day Patient Representative Workshop so they can receive training that will allow them to help the FDA meet its critical responsibility of guiding the development and evaluation of safe and effective medical products.

Among these representatives were caregivers who have a personal experience with Batten disease. Sadly, each lost a young son to the disease. But in the face of this tragedy, these two mothers have advocated tirelessly to find a cure for this disease and worked to educate other parents, much like the way that our foundation was started. Here at BBDF we believe in finding hope in the face of this disease. We believe in working tirelessly to find a treatment for Batten disease and other rare diseases in the process. To read the full article on the FDA’s blog click HERE!


Batten disease on Good Morning America