Batten Disease Research- $117 Honorarium Provided

As an organization committed to supporting Batten disease patients and their families, we are writing to bring your attention to a research study that seeks to understand the challenges faced by patients and families impacted by Batten.

Beyond Batten Disease Foundation was established to eradicate juvenile Batten disease by raising awareness and funds to accelerate research for a treatment or cure. We are collaborating with Nita Patel of Amicus Therapeutics, other Batten disease related patient organizations and Engage Health (a health research firm), on this important study. Results from this study will be submitted for publication in order that others can learn from your experiences.

We hope you will consider participating! To be eligible you must be:

  • A person diagnosed with a CLN1, CLN3 or CLN6 variant of Batten disease who is age 18 years or older OR
  • Their parent/legal guardian*
  • Diagnosis of a CLN1, CLN3 or CLN6 variant of Batten disease by provision of a proof of disease form
  • Able to read, write and communicate in English or German
  • Able to grant informed consent
  • Willing to complete a survey and RSVP, and to participate in a 50-minute telephone interview
  • Ability to view or receive a document from the interviewer before or during the interview (web browser, ability to receive a text, fax or document by mail or email)

*Please note: A parent whose child has passed on in the last 5 years is able to participate in the Survey/RSVP. Only one family member will be eligible to complete the RSVP and interview for one patient. If a family has more than one person with Batten disease, one family member may be eligible to complete the RSVP and interview for each patient.

Involvement consists of filling out a Survey/RSVP, answering a few short questions to ensure you qualify, and providing a document that shows proof of disease (a school note, copy of chart note or another document). This will take about 10 minutes. You will then be eligible to participate in a 50-minute telephone interview conducted in English. For your time, you will be paid a $117 honorarium if you complete the interview. No preparation is required to participate, but you will need to be at a computer.

If you would like to participate please:
Go to Understanding the Challenges Faced by Patients and Families Impacted by Batten Disease Through the Eyes of Patients to fill out the survey and RSVP with dates and times that you are available. Please note that times outside of standard business hours are available.
At the screen, please check the box that says “Check if you have no code” to proceed to the RSVP
If you are unable to access the RSVP and survey from the link above, please copy and paste the following URL into your browser bar to access it: https://www.engagehealth.com/survey/TakeSurvey.aspx?SurveyID=72KI4m3K
Once you have completed the survey and RSVP, Engage Health will contact you to confirm the date and time of your telephone interview, or to notify you that the interviews are full, if that is the case.

If you would like to forward this invitation to other families living in the United States who are impacted by Batten disease, please do so, but remember that the interviews are being conducted on a very limited basis, and participation is first come, first served. Please respond quickly if you would like to participate!

Thank you for your interest,

Mary Beth Kiser
President and CEO

Hope on the Green 2021

For the past 12 years we have been blessed with phenomenal weather, enthusiastic golfers, and energetic volunteers. Yesterday was no exception, we gathered once again in celebration of HOPE, with our collective mission in mind… #SaveWill . Thank you to everyone who joined us at our 2021 HOPE on the Green. We look forward to sharing our recap photos online this week!

Thank you to our Photography sponsor:

Hughes & Cozad Orthodontics

Run to the Sun 2021 Thank You!

A note from Austin Youth Fitness:
“Thanks to everyone who participated in the Run to the Sun and David Phillips Memorial Mile! Together we raised $4,010 for the Beyond Batten Disease Foundation!

If you haven’t made a donation yet, there’s still time to do so here!
I am working on distributing shirts, but still need some orders for the online store in order for them to be finalized. If you registered after February 20th, you did not officially get a shirt and will need to order one here.

Congrats to our top fundraiser, Felix Statler who raised $740!!! Felix also clocked his mile time at 7:50!

We hope you keep running, keep living life to the fullest, and have a great rest of your semester and 2021! Brighter days ahead where we can all Run to the Sun together!”

BBDF Announces Agreement with Actelion Pharmaceuticals Ltd to Provide Drug Product for BBDF-101

Austin, Texas, March 2, 2021

Beyond Batten Disease Foundation (BBDF) is pleased to announce an agreement with Actelion Pharmaceuticals Ltd, a Janssen Pharmaceutical Company of Johnson & Johnson, to provide Zavesca® (miglustat) for the development of BBDF-101, a proprietary combination of miglustat and trehalose to treat juvenile Batten disease (CLN3). Janssen will provide drug supply for the clinical trials and Extended Access Program, and right of reference to data within the Zavesca New Drug Application (NDA) to support the development of BBDF-101.

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BBDF and Theranexus collaborate to publish journal

BBDF and Theranexus collaborate to publish “Evaluating and modulating TFEB in the control of autophagy: toward new treatments in CNS disorders” in the journal Fundamental & Clinical Pharmacology

Intense scrutiny of patient cells and animal models of CLN3 disease over the past 3 decades demonstrates that the loss of CLN3 protein impacts multiple cellular functions. The majority of dysfunction is in cellular compartments responsible for cellular recycling and waste management. This review, co-authored by Beyond Batten Disease Foundation and Theranexus, describes how drug-mediated activation of transcription factor EB (TFEB) clears toxic waste from Batten and other neurodegenerative disease models and describes ongoing pharmaceutical plans to use TFEB activators to treat these diseases.

View here

BBDF-101 Announcement

We are excited to announce the following major milestone for BBDF!

The FDA has awarded Orphan Drug and Rare Pediatric Disease designations to Beyond Batten Disease Foundation for BBDF-101. The aim of the program is to facilitate the development of new drugs and biological products for the prevention and treatment of rare pediatric diseases. These designations provide accelerated review for approval, support with the FDA regulatory process and at least seven years of post-approval protection, as well as exemption from filing fees that normally have to be paid to the FDA. These designations do not impact the clinical trial, but are beneficial once the therapy receives New Drug Approval. We are grateful to Theranexus for their continued partnership and commitment to initiating a clinical trial for BBDF-101 as quickly as possible.

THERANEXUS AND BBDF OBTAIN ORPHAN DRUG DESIGNATION (ODD) AND RARE PEDIATRIC DISEASE DESIGNATION (RPDD) FROM THE FOOD AND DRUG ADMINISTRATION (FDA) FOR BBDF-101 FOR BATTEN DISEASE

  • Orphan Drug Designation (ODD) is a status that provides seven years of additional post-approval protection and exemption from filing fees
  • Rare Pediatric Disease Designation (RPDD) qualifies the sponsor at the time of registration for a salable, transferable priority review voucher which can be used to speed up the approval process for another drug candidate

Lyon, August 28, 2020 – Theranexus, a biopharmaceutical company innovating in the treatment of neurological diseases, and Beyond Batten Disease Foundation (BBDF) today announced the decision by the Food and Drug Administration (FDA) to award Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) to the drug candidate BBDF-101 for Batten disease, a rare, fatal, genetic disorder of the nervous system for which there is no treatment.

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BBDF-101 Announcement

We are excited to announce the following major milestone for BBDF! Theranexus, a publicly-traded European pharmaceutical company, has committed the $20 million and expertise necessary to complete the clinical trial and commercialization for our drug discovery, BBDF-101.

Theranexus and Beyond Batten Disease Foundation (BBDF) announce the signing of a worldwide exclusive license for Batten disease drug candidate BBDF-101
  • The agreement covers the development and marketing of the drug following a single clinical trial due to begin in 2020. 
  • If successful, this trial will lead directly to the drug’s approval.

Lyon, 12 December 2019 – Theranexus, a biopharmaceutical company innovating in the treatment of neurological diseases and pioneer in the development of drug candidates modulating the interaction between neurons and glial cells, is pleased to announce the signing of an agreement with Beyond Batten Disease Foundation, granting it a worldwide exclusive license to develop and commercialize the drug candidate BBDF-101 for juvenile Batten disease.

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2nd Annual Bind Up Batten Golf Charity

Bind Up Batten brought together the community on July 20, 2019, for the 2nd Annual Batten Disease Golf Charity Outing. Over 80 golfers came out on a beautiful, cloudy day in Waterville, Ohio to raise $12,700 for juvenile Batten Disease research.

In addition to the golfers, Bind Up Batten would like to thank the families, friends, musicians, Fallen Timbers Fairways and all the sponsors who provided their support to help make this event happen.

Special thanks and recognition to Mike’s parents TJ and John Klein as well as Donna and Paul Iwinski for their incredible effort along with many other volunteers who made this day possible.

Together we can Bind Up Batten!

Watch the video

We are Austin Interview with Garland Benson

We Are Austin did an interview highlighting Garland Benson and the Be a Hero Campaign

Check out his interview here:

If you had a goal to raise one million dollars in two years, you might think it would be impossible! Now imagine you’re 12 years old and setting out on this incredible mission. Well, Garland Benson did just that, and his perseverance will help fund research to treat his older sister’s degenerative disease. Garland is here this morning to talk about his remarkable success raising funds for Beyond Batten.

Thanks again to everyone who helped Garland hit his goal!

BBDF is actively pursuing a pharmaceutical partner to support the clinical trial and commercialize the treatment.  Donations are rolling in and every additional dollar raised by Garland’s campaign will help offset activities required by the FDA to start the trial.

Donate today.

People Magazine: Garland’s One Million Dollar Story

People Magazine has just published an article highlighting Garland’s quest to raise a million dollars for his sister.

Thanks again to everyone who helped Garland hit his goal!

BBDF is actively pursuing a pharmaceutical partner to support the clinical trial and commercialize the treatment.  Donations are rolling in and every additional dollar raised by Garland’s campaign will help offset activities required by the FDA to start the trial.

Donate today.

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