BBDF Supports Project Baby Dillo!
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:
*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
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!”
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.
Manuscript by da Costa, Anaelle; Metais, Thibaud; Mouthon, Franck; Kerkovich, Danielle; Charvériat, Mathieu, was successfully accepted by the journal “Fundamental & Clinical Pharmacology.”
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
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.
The FDA has awarded Orphan Drug and Rare Pediatric Disease designations to Beyond Batten Disease Foundation for BBDF-101.
BBDF’s principal scientist, Danielle Kerkovich, recieves recognition for her paper as one of the most read in Molecular Genetics & Genomic Medicine.
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.