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) https://beyondbatten.org and Brain Canada http://braincanada.ca 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 www.beyondbatten.org.

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.  www.ncl-foundation.com

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 Baby.com, 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 http://www.cdbaby.com/cd/emilygimble12 , Apple’s iTunes Store or on Amazon.

The New York Stem Cell Foundation Partners with Beyond Batten Disease Foundation to Fight Juvenile Batten Disease

Batten Disease Support and Research Association collaborates to give families the opportunity to participate at its Annual Family Conference

NEW YORK, NY (July 23, 2014) – The New York Stem Cell Foundation (NYSCF) and Beyond Batten Disease Foundation (BBDF) have partnered to develop stem cell resources to investigate and explore new treatments and ultimately find a cure for juvenile Batten disease, a fatal illness affecting children.

NYSCF scientists will create induced pluripotent stem (iPS) cell lines from skin samples of young people affected by juvenile Batten disease as well as unaffected family members. IPS cell lines are produced by artificially “turning back the clock” on skin cells to a time when they were embryonic-like and capable of becoming any cell in the body. Reprogramming juvenile Batten iPS cells to become brain and heart cells, will provide the infrastructure needed to investigate what is going wrong with the cells adversely affected by the disease. Thus far, efforts to study juvenile Batten disease have been done using rodent models or human skin cells; neither of which accurately mimic the disease in the brain, leaving researchers without proper tools to study the disease or a solid platform for testing drugs that prevent, halt, or reverse its progression. This will be the largest and first genetically diverse collection of human iPS cells for a pediatric brain disease.*

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Beyond Batten Disease Foundation Announces Participation in 2nd Annual “Amplify Austin” Event

In 2008, local Austin couple Charlotte and Craig Benson received the devastating diagnosis that their then five-year-old daughter, Christiane, had juvenile Batten disease. Batten is a rare, inherited neurological disorder that strikes young children, first causing vision loss and seizures, then cognitive and motor impairment, and ultimately late death by the late teens or 20s. Determined to make a difference, the Benson family established Beyond Batten Disease Foundation (BBDF). The Bensons experienced an outpouring of love and support from their friends and family in the Austin community. Over the past five years, these supporters have continued to give their time, talent and treasure to help the foundation accomplish its mission of eradicating Batten disease. Because of their support, BBDF has been able to make huge strides towards its goal – raising funds and awareness for research and leading development of an easy and inexpensive, groundbreaking blood test to detect the gene mutations that cause Batten disease as well as 700-plus other rare, but serious and often fatal childhood ailments

Beyond Batten Disease Foundation has strong local ties in the Austin community and is proud to announce its participation in Amplify Austin 2014. What began as the inspiration of one child has grown to be the voice for all affected children and their families. BBDF looks forward to accelerating their mission with the help of Amplify Austin and the Austin community. To make a contribution to BBDF through Amplify Austin, schedule a donation today or log on to www.amplifyatx.ilivehereigivehere.org/BeyondBatten on March 20th at 6 p.m. through March 21 at 6 p.m. To learn more about BBDF, visit www.beyondbatten.org

ABOUT AMPLIFY AUSTIN: Last year, the “Amplify Austin” event, powered by Kimbia, raised nearly $3 Million for Central Texas nonprofits in only 24 hours. This hugely successful event benefitted over 300 participating local nonprofits. In today’s changing environment for nonprofits, development executives and community leaders are turning to online-enabled giving days for fresh and highly effective ways to: attract new donors and more funds; drive greater collaboration with nonprofit organizations; and raise community-wide awareness of their work, value and brand. Amplify Austin is an initiative of I Live Here, I Give Here. To learn more, visit www.amplifyatx.ilivehereigivehere.org

To view this release on PR Web, click here.

Going social for Alzheimer’s research

by Jim Cirigliano in Drug Discovery News

published January 10, 2014

NEW YORK—The Alzheimer’s Drug Discovery Foundation has announced a new program that will provide a searchable online marketplace for academic and small biotech researchers seeking contract research organizations (CROs) that provide drug discovery and development services. The program, called ADDF ACCESS, links small biotech and academic research communities to a network of more than 130 CROs that provide industry standard services for central nervous system (CNS) indications.

The program also includes access to drug discovery experts who can provide help in selecting from among the CROs, as well as guidance on the drug discovery process. Registration for the ACCESS program is free.

“The ADDF is regularly approached by scientists in academia and small biotech to provide recommendations for contract research organizations and to provide advice in navigating this sector of the life sciences industry,” says Dr. Rachel Lane, assistant director of scientific affairs at the Alzheimer’s Drug Discovery Foundation. “ADDF ACCESS provides a go-to resource of CROs working in the CNS space and provides guidance on selection and management of CROs.”

CROs have become vital partners for drug discovery and clinical development among programs conducted by academia and small biotech firms that often run on virtual models, Lane notes. CROs are able to offer external validation of data, clinical lab environments that are industry regulated, and drug development expertise.

In addition, CROs have proven valuable to the pharmaceutical industry and private equity groups who use them. Large pharmaceutical companies have developed the means of identifying and managing CRO networks of their own, but academia and small companies typically lack access to these networks or lack the experience to navigate them or use their services effectively.

In addition to providing academic and small biotech researchers access to a selection of CROs, the ADDF program has also negotiated discounted pricing with several of the vendors in the network. Some CROs in the network will provide their services at discounted rates to investigators who are referred through the ACCESS program.

Although the marketplace is not currently a list of “preferred” vendors, the ADDF and a team of consultants are working to perform due diligence on the CROs included in the network. The ADDF has created educational materials designed to guide researchers through best practices for selecting a vendor and managing a CRO contract.

The ADDF is also working to expand the network in the future and has begun building a consortium of nonprofit partners that includes scientists working on many neurodegenerative diseases. BrightFocus Foundation and Beyond Batten Disease Foundation have provided partnership support for the program’s expansion so far, and the ADDF hopes to reach an even broader, multidisciplinary community.

The next step for the program is the completion of its social media platform—a community feature of the site that will allow researchers to relate their experiences with the network of CROs to one another and to engage in an online forum.

“The community feature will provide an online discussion platform and social networking tool to allow users to provide objective feedback on CROs, facilitate discussions on emerging trends in drug discovery for neurodegenerative diseases, allow users to follow companies and create networks of researchers to promote the formation of interdisciplinary teams across CNS therapeutic areas,” says Lane.

The ADDF also looks to continue doing due diligence on the companies in its network to ensure that they are properly categorized according to their core competencies. Looking somewhat farther into the future, the ADDF anticipates developing a platform to highlight promising drug discovery programs within the ADDF portfolio that are available for partnering or licensing opportunities.

The Alzheimer’s Drug Discovery Foundation’s mission is to accelerate the discovery of drugs to prevent, treat and cure Alzheimer’s disease, related dementias and cognitive aging. The ADDF provides funding for drug discovery and clinical development programs in academia and biotechnology companies via a venture philanthropy approach, with funding spanning the translational space from preclinical development through Phase 2 clinical trials.

The ADDF regularly partners with other foundations and industry to leverage resources and funding for its programs. The ADDF has granted nearly $60 million to fund 400 Alzheimer’s drug discovery programs and clinical trials in academic centers and biotechnology companies in 18 countries.


Overnight run from Enchanted Rock to Austin to Save Lives

How far would you run to save a life? Ten miles? 26.2? Even 50? In late April, 25 teams of runners will run more than 90 miles in a spectacular overnight journey to raise money and awareness for juvenile Batten disease, a rare, fatal, neurodegenerative disease that affects children.

Run to the Sun Relay is an overnight, long distance relay that starts each year at Enchanted Rock State Park and ends at Murchison Middle School in Austin, Texas. The 4th Annual Run to the Sun Relay will begin on April 26, and will end with a sunrise celebration and 2K Fun Run on April 27. We expect over 400 people to participate. We encourage all participants and their families, and friends of Beyond Batten Disease Foundation to join us at the sunrise celebration breakfast held at the Murchison Middle School track. We will stand together at sunrise to acknowledge the strength of all those affected by Batten disease and envision a world where this devastating disease no longer exists.

Run to the Sun was created four years ago by the parents of an Austin fifth grader with juvenile Batten disease. Each year, her teachers and family friends form teams to run, and her friends from school work as volunteers during the race. This is a remarkable story – about an unusual race and an unusual family, who responded to this devastating diagnosis by working to make a real difference.

Over the past 3 years, the relay has raised almost $700,000 to support Batten disease research. This year, the foundation wants to increase our total to $1 million, and needs the support and participation of the Austin and Batten disease communities to reach that goal. Whether old or young, fast or slow, near or far, this event offers a variety of opportunities for both runners and non-runners to get involved in the fight to eradicate Batten disease.

WE WOULD LOVE YOUR HELP IN PUBLICIZING SIGNUPS FOR THIS EVENT NOW, AND IN COVERING RUN TO THE SUN ON THE DAY OF THE RELAY. We’re looking for runners to participate in the relay and for volunteers to support them as they run through the night. Our 2K Fun Run is open to people of all ages and speeds. This is a fantastic opportunity for many kinds of coverage, including a fun way to keep New Year’s fitness resolutions, a medical story, an education story about the involvement of local teachers, or a live shot on the night of April 26 or the morning of April 27.

To learn more about Run to the Sun, please visit www.runtothesunrelay.com or contact Mary Beth Kiser at info@beyondbatten.org or 512-275-2600. We are happy to connect you with runners, with volunteers and with families affected by juvenile Batten disease.

For more information about juvenile Batten disease and Beyond Batten Disease Foundation, please visit www.beyondbatten.org.

Click here to watch the inspirational recap video from Run to the Sun Relay 2013.

To view this release on PR Web, click here.

Alzheimer’s Drug Discovery Foundation Expands its ADDF ACCESS Program to Improve Quality and Efficiency of Drug Research for Neurodegenerative Diseases

NEW YORK, NY, November 6, 2013 – The Alzheimer’s Drug Discovery Foundation (ADDF) announced yesterday the launch of its newly expanded ADDF ACCESS program to provide scientists in academia and small biotechnology companies with access to a virtual network of drug discovery experts and contract research organizations (CROs) that have experience developing therapies for neurodegenerative diseases. Through the effective selection of CROs and use of their services, researchers may be able to accelerate their research and bring novel therapies to patients faster.

“The ADDF is excited to expand the availability of this valuable resource for the scientific community,” said Howard Fillit, MD, executive director and chief science officer of the ADDF. “Our mission at the ADDF is to accelerate the discovery and development of new Alzheimer’s drugs. Drug discovery is a true interdisciplinary effort and ADDF ACCESS seeks to connect scientists with the diverse range of resources and services they need to expedite their research.”

ADDF ACCESS now gives academic and small biotechnology research communities access to a network of more than 130 CROs and the services they provide. The expanded and redesigned portal allows users to customize their search for CROs and access a virtual network of drug discovery experts who can provide guidance on CRO selection and research design.

To make this resource available to the broader community and promote collaboration between scientists working on different neurodegenerative disease, the ADDF is building a consortium of nonprofit partners. BrightFocus Foundation and Beyond Batten Disease Foundation (BBDF) have already provided support for the expansion of ADDF ACCESS.

“When budgets are tight, efficient networking and collaborations are key to research successes,” said Guy Eakin, PhD, vice president of scientific affairs at BrightFocus Foundation. “We’re proud to partner with ADDF to accelerate Alzheimer’s drug research through the sharing of critical resources.”

Danielle Kerkovich, PhD, principal scientist of BBDF, said, “We are thrilled to be a part of ADDF ACCESS, helping close the gap between discovery and treatment. With so many Americans, both young and old, facing the threat of serious neurodegenerative diseases, we must bring together the brightest minds, highest quality of resources, and the most experience to save time, money, and lives.”

The expansion of ADDF ACCESS was launched in conjunction with the 5th annual Partnering for Cures meeting in New York City. The ADDF ACCESS program was selected as an Innovator Presentation and was presented on Tuesday, November 5, 2013 10:45 – 11:10 AM EST.

About the Alzheimer’s Drug Discovery Foundation (ADDF)

The mission of the Alzheimer’s Drug Discovery Foundation (ADDF) is to accelerate the discovery of drugs to prevent, treat and cure Alzheimer’s disease, related dementias and cognitive aging. The ADDF has granted nearly $60 million to fund 400 Alzheimer’s drug discovery programs and clinical trials in academic centers and biotechnology companies in 18 countries. For more information, please visit www.AlzDiscovery.org.

Press Contact:

Michael Grela: +1 212 594 5500 or mgrela@KYNE.com

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