Beyond Batten Disease Foundation announces Craig Benson’s appointment to the NRI International Advisory Board

Austin, TX – July 15, 2015 – Beyond Batten Disease Foundation (BBDF) is pleased to announce Craig Benson’s appointment to the Jan and Dan Duncan Neurological Research Institute (NRI) International Advisory Board. Consistent with BBDF’s strategy and goals to connect our research with top-ranked institutions, Craig is eager to join the NRI in its mission.

The NRI at Texas Children’s Hospital aims to reduce the temporal and conceptual gap between initial gene discovery and clinical applications by providing the context, core services, and community to support interdisciplinary collaborations between physicians, scientists, and patients. Designed to advance the understanding of neurological disorders, such as Batten disease, through basic and translational research and ultimately mitigate the impact of these devastating disorders on human life, the NRI has been integral in BBDF’s success.

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Beyond Batten Disease Foundation Renowned Italian Researcher Publishes Groundbreaking Research in Top International Scientific Publication

Austin, Texas – June 9, 2015 – Beyond Batten Disease Foundation (BBDF) announces groundbreaking research published in Nature Cell Biology. The lysosome biology research is leading the way to a cure for juvenile Batten Disease.

“We think this research is our strongest candidate for therapeutic development for juvenile Batten Disease and understanding lysosome biology is the way to find a cure for this devastating disease,” said Danielle M. Kerkovich, PhD, BBDF Principal Scientist.

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Beyond Batten Disease Foundation Joins Innovative Network of Venture Philanthropies

Austin, Texas – May 12, 2015 – Beyond Batten Disease Foundation has become a participating organization of The Research Acceleration and Innovation Network (TRAIN), a program of FasterCures, bringing the total number of venture philanthropies in TRAIN to 80.

FasterCures established TRAIN in 2005 to connect innovators in the disease research space with the vital resources, tools and relationships to catalyze development of new therapies and cures. Through the network, innovation in one disease area is translated to another in order to achieve treatment breakthroughs for all.

“We are pleased to expand our fight against juvenile Batten disease by becoming a partner in TRAIN to transform juvenile Batten disease research and accelerate our timeline to a cure,” said Danielle M. Kerkovich, PhD, BBDF Principal Scientist.

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Beyond Batten Disease Foundation and the New York Academy of Sciences Release a New Podcast Revealing Innovative Ways to Cure Rare Disease

Austin, Texas – Beyond Batten Disease Foundation (BBDF) and the New York Academy of Sciences (NYAS) are working together in a new and innovative way to find a cure for a rare disease. While diseases like cancer and Alzheimer’s are widely known, rare diseases like juvenile Batten lack the attention and resulting funds needed to research, develop, and implement treatments. Juvenile Batten even lacks awareness among the medical and research community itself.

To raise awareness among research thought leaders and focus their collective energies on a key topic in juvenile Batten disease research, BBDF Principal Scientist, Danielle Kerkovich, PhD, proposed a unique think-tank of top experts from the Parkinson’s, Alzheimer’s, cancer and Lysosomal Storage Diseases (LSDs) fields to focus on our common drug target—a cell’s lysosome. Lysosomes are the recycling centers of the cell playing vital roles in the removal of cellular waste and keeping critical cell systems in balance. In diseases like juvenile Batten, other LSDs and adult forms of neurodegeneration, lysosome dysfunction compromises multiple cell systems eventually resulting in cell death.

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Platform for Engaging Everyone Responsibly Awardees Announced

WASHINGTON, DC – March 24, 2015 – Today Genetic Alliance announced the fifteen awardees for Phase I of its initiative to create a ‘white label’ of the Platform for Engaging Everyone Responsibly (PEER). PEER enables individuals to share health information with researchers and each other on their own terms to advance an understanding of health and disease, and to accelerate the development of cohorts for clinical trials. The fifteen selected awardees will work with Genetic Alliance to develop and refine the ‘white label’ PEER while engaging their communities.

Beyond Batten Disease Foundation (BBDF) was one of the fifteen selected awardees. BBDF plans to establish a functional network of existing and emerging Batten registries. Integrating existing registries to standardize and maximize data will provide academic investigators, clinician scientists, Pharma and clinical trialists a critical mass of informative data, accelerate the path to clinical trials, stimulate new research leading to new scientific insights, and provide a platform for patients and families to share their experiences, concerns and wishes.

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Where There is a Will, There is a Way! Charities Partner at The Woodlands Marathon to Bring Hope to Children on World Rare Disease Day

THE WOODLANDS, TEXAS – Feb. 28. – Where there is a Will, there is a way! Meet the inspirational Will Herndon and his mother Missy at the Woodlands Marathon Saturday, February 28, 2015. Will’s Warriors will run on World Rare Disease Day to support Will, who is fighting a rare disease.

Six years ago, Will and his family received a devastating diagnosis. Will, a loving, bright, energetic 6-year-old, has juvenile Batten disease—a rare, genetic, neurodegenerative disorder. Batten disease attacks an initially healthy child and causes vision loss, loss of cognitive skills, and seizures. Progressively, children suffer loss of memory and speech until they are mentally and physically incapacitated, eventually leaving them wheelchair bound, and then bedridden. With no current treatment or cure, Batten disease is always fatal, often by the late teens or early twenties. Will, now 12, is blind. His family is heart-broken but driven to find a cure.

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Beyond Batten Disease Foundation of Texas and Brain Canada Launch New Partnership to Expand Network of Researchers Internationally

Austin, Texas– February 4, 2015 – Beyond Batten Disease Foundation (BBDF) and Brain Canada are pleased to announce a new partnership that will hire researchers in Canada to investigate, explore and find a cure for juvenile Batten disease.

The research project is for $1.5 million over three years. The goal is to further grow the network of international researchers working to find a cure for juvenile Batten Disease. The deadline to submit a letter of intent is February 9, 2015.

“We are pleased to expand our fight against juvenile Batten disease to experts in Canada to transform juvenile Batten disease research and accelerate our timeline to a cure,” said Danielle M. Kerkovich, PhD, BBDF Principal Scientist.

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Beyond Batten Disease Foundation and the New York Stem Cell Foundation Chosen as a National Innovator by the Milken Institute as They Partner to Find a Cure for Juvenile Batten Disease

The Battle is Personal for a Texas Father Who is Trying to Save His Own Daughter’s Life

New York City, New York – November 17, 2014 – Beyond Batten Disease Foundation (BBDF) and the New York Stem Cell Foundation (NYSCF) have been selected as a national innovator by the Milken Institute and will present their breakthrough findings about juvenile Batten disease at the 6th annual Partnering for Cures, November 16-18 in New York City.

Craig and Charlotte Benson established Beyond Batten Disease Foundation in August 2008 after their then five-year-old daughter, Christiane, was diagnosed with juvenile Batten disease.  Together with hundreds of families affected by Batten disease, and many more supporters who share their hope and resolve, they are working tirelessly to create a brighter future for Christiane, and all children with Batten disease.

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Beyond Batten Disease Foundation partners with NCL Foundation to award $344,742 to research team of Dr. Marco Sardiello, Baylor College of Medicine, Houston

As part of a larger $1.75M grant, BBDF continues to support Dr. Marco Sardiello’s research, investigating the role of CLN3 the juvenile Batten disease gene, CLN3, in Juvenile Neuronal Ceroid Lipofuscinosis (JNCL).

Recognizing the promise of BBDF-funded research, NCL Foundation in Hamburg, Germany, awarded the 5th NCL Research Award to Dr. Marco Sardiello and his team at Baylor College of Medicine in Houston, Texas. The award, which amounts to $133,942, will finance a postdoctoral fellow, Dr. Alberto diRonza, whose work will be dedicated to unraveling the primary role of the normal CLN3 protein and the lysosomal defects that result in Batten disease. NCL’s funding is in conjunction with BBDF’s funding of $210,800 to Dr. Marco Sardiello and his team.  With these combined funds, the researchers hope to gain insight into the role of CLN3.

Dr. Marco Sardiello and his team are dedicated to the research and development of innovative therapies to treat lysosomal diseases (LDs), including juvenile Batten disease. LDs are the most common childhood neurodegenerative diseases. The majority of LDs are caused by defects in one of over 60 known soluble lysosomal enzymes or 25 transmembrane proteins in the outer layer of lysosomes. Lysosomes are cellular organelles that play a key role in the degradation and recycling of cellular organelles, proteins, lipids and other substances. Therefore, defects in genes that encode lysosomal proteins result in excessive accumulation and devastating effects.

Complicating treatment, the normal CLN3 gene (when mutated, causes juvenile Batten disease) encodes a transmembrane protein. Therefore, candidate therapies targeting soluble enzyme deficiencies, such as enzyme replacement therapy, bone marrow transplantation, or gene therapy, are generally not considered for treating juvenile Batten.

Dr. Sardiello and his team are investigating transcription factor EB (TFEB). Dr. Sardiello was part of a team which discovered TFEB is a master gene in the network regulating the biogenesis and activity of lysosomes and that inducing TFEB can increase lysosomal clearance in certain disorders. This discovery and its continued study show potential in the treatment of juvenile Batten disease.

About Beyond Batten Disease Foundation
Beyond Batten Disease Foundation (BBDF) is the world’s largest organization dedicated to funding research for a treatment or cure for juvenile Batten disease. Batten disease is a rare, inherited pediatric neurological disorder, which begins with vision loss and seizures, followed by cognitive and motor impairment, and ultimately death by the late teens or 20s. Since its inception in 2008, BBDF has raised over $14.6 million for research through donations, co-funding, leveraging and partnerships. BBDF is spearheading a unique, cohesive strategy, incorporating independent scientific resources and collaboration with related organizations to drive research in Batten Disease. For more information, visit

About NCL Foundation
The NCL Foundation was founded 2002 by Dr. Frank Husemann, after his then 6-year-old son Tim was diagnosed with JNCL. Neuronal Ceroid Lipofuscinosis is a rare metabolic disorder which leads to a progressive loss of nerve cells. It is the most common form of childhood dementia. Affected children suffer from neurodegeneration affecting different types of neurons, also in the retina, and this results in early blindness, mental deterioration, loss of motor function and the development of epileptic seizures. Many NCL patients do not survive past their third decade.

Press contact: Mary Beth Kiser, President  & CEO, Beyond Batten Disease Foundation, 512.275.2600

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Emily Gimble Launches New Single for Beyond Batten Disease Foundation

Pianist, vocalist and newest member of Asleep at the Wheel, Emily Gimble has a brand new single, “Mighty Like a Rose,” currently available for downloads on the web at CD, iTunes, Amazon and other online distributors. All proceeds benefit Beyond Batten Disease Foundation, a non-profit organization established to eradicate Batten Disease. Often celebrated for her raise-the-roof voice and rambunctious keyboard stylings, Gimble shows her more tender side in this reworking of the 1901 lullaby by Nevin and Stanton. Produced by Floyd Domino, who also takes over on piano, Gimble is accompanied by Ryan Gould on upright bass, J. D. Pendley on guitar and Jonathan Doyle on clarinet.

“This is a most unusual gift for our foundation and has already proved to be a unique fundraiser. For a small amount of money, Emily’s fans can add this gorgeous rendition to their music library and help us spread awareness and research funds for a cure for Batten disease,” said Mary Beth Kiser, president and CEO of the nonprofit foundation.

The musical connections in this story are numerous and unexpected. Emily is the daughter of musician, recording artist and music teacher Dick Gimble and granddaughter of Grammy-winning Texas swing fiddler Johnny Gimble, who played with Bob Wills. Al Strickland also played with Wills and coincidentally taught the finer points of swing piano to Floyd Domino, two-time Grammy winner with Asleep at the Wheel. Additionally, “Mighty Like a Rose” was recorded Bismeaux Studio, which is owned by Ray Benson, Emily’s new boss.

“My mom sang this to me when I was a baby. Her mother sang it to her and I sang it to my two boys,” laughs Mary Beth as she recalls the history of the lullaby in her family. While mastering the song at Terra Nova Digital Audio, owners Jerry and Diane Tubb both remarked that it rang a bell. “That brings back fond memories to me,” said Jerry. The song has been covered over the years but has not sounded so good not since Frank Sinatra’s version from the forties. Floyd and Emily were able to capture the essence of the Harry James classic recording.

Beyond Batten Disease Foundation was established in 2008 by Craig and Charlotte Benson after their five year old daughter, Christiane, was diagnosed with juvenile Batten disease, a rare and fatal inherited disorder that usually begins in childhood. The first symptom is progressive vision loss, followed by personality changes, behavioral problems and mobility issues. Seizures can begin at any time during the course of the disease and tend to worsen with age. There is no treatment or cure.

All proceeds go to the foundation in its quest to find a treatment and a cure for this heartbreaking disease. “Mighty Like a Rose” with vocal by Emily Gimble and an instrumental version can be downloaded at , Apple’s iTunes Store or on Amazon.

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