When the foundation was created in 2008, there was very little known about juvenile Batten disease. With no known treatment and no cure, we had a long and uphill battle ahead of us. Thanks to the continued support of the community and generous donors, the foundation has made great progress towards our goal of eradicating Batten disease. Here are just a few of our proudest accomplishments.
August 1
The foundation is established
The foundation is established by Charlotte and Craig Benson in August after their then five-year-old daughter, Christiane, is diagnosed with juvenile Batten disease.
October 10
Genetic program discovered
World-renowned Italian researches Andrea Ballabio, MD, PhD, and Marco Sardiello, PhD, discover a genetic program that controls the functions of lysosomes, the cells recycling centers damaged in JNCL. February 28
Batten and Rare Disease Day named in Texas
Governor Rick Perry names February 28, 2009 Batten and Rare Disease Day in Texas. 
March 26
Missy and Wayne Herndon establish the Will Herndon Fund
Missy and Wayne Herndon establish the Will Herndon Fund of the Beyond Batten Disease Foundation to join forces in raising funds for a cure after their son, Will, is diagnosed with JNCL. 
April 26
Craig Benson joins BDSRA board
Craig Benson joins the board of Batten Disease Research and Support Association (BDSRA) to promote partnerships and further collaboration efforts with affected Batten families. 
May 26
The foundation hires Danielle M Kerkovich, PhD
The foundation hires Danielle M Kerkovich, PhD, a trained neurobiologist with extensive experience building research programs in the federal funding and nonprofit sectors. Dr. Kerkovich determins the “State of Science” in JNCL and identifies gaps in our understanding to design plans to drive therapeutically relevant discovery to its necessary conclusion: clinical trials in JNCL. June 26
Cherie and Jim Flores give largest gift in Batten disease history
Cherie and Jim Flores donate $2 million and BBDF contributes $500,000 to establish laboratories for Drs. Ballabio, Sardiello and their colleagues at the Jan and Dan Duncan Neurological Research Institute of Texas Children’s Hospital. July 24
BBDF co-sponsors a conference for drug discovery in Batten disease
The foundation, in partnership with the Alzheimer’s Drug Discovery Foundation, Batten Disease Support and Research Association, and NCL-Stiftung sponsors a conference focused exclusively on drug discovery in Batten disease. This meeting forges new collaborations between academic scientists and drug discovery experts who turn research findings into therapy. 
April 26
BBDF co-funds the National Center for Genome Resources
BBDF co-funds the National Center for Genome Resources to develop a rare disease genetic test panel to prevent and diagnose JNCL and hundreds of other devastating genetic illnesses in children. November 26
BBDF supports the Cell Biology of the Neuron Gordon Research Conference
BBDF supports the Cell Biology of the Neuron Gordon Research Conference (GRC), where leading investigators from around the globe discuss their latest work. BBDF’s support contributes to the critical evaluation of data on cells affected by juvenile Batten disease. 
April 26
BBDF hosts the inaugural Run to the Sun Relay
BBDF hosts the inaugural Run to the Sun Relay. The event was inspired by Lance Thompson, who continues to chair the relay annually. His dedication and support allow the event to be an ongoing fundraising success. 
June 1
The foundation delivers a grant to King’s College, London
The foundation delivers a grant to King’s College London researchers Drs Jonathan Cooper and Brenda Williams to explore glial brain cells as novel drug targets in JNCL. 
July 26
Clinical trial for carrier screening test completed
Dr. Kingsmore and colleagues at Children’s Mercy Hospital in Kansas City complete a clinical trial to validate the sequencing-based carrier screening test developed through BBDF funding. The number of childhood diseases the test can screen has grown from 448 to 620. 
August 26
Craig Benson is interviewed on NPR
Craig Benson, founder of BBDF and initiator of the recently published rare disease genetic test, is interviewed on nationally syndicated NPR, a network of 900 public radio stations. Listen to the Story » February 9
The foundation co-hosts its first scientific conference
Beyond Batten Disease Foundation hosts its first scientific conference on Feb. 9, 2011, in San Diego, California, in partnership with the Alzheimer’s Drug Discovery Foundation, the Batten Disease Support and Research Association, and NCL-Stiftung. 
September 26
BBDF co-hosts a symposium in Germany for doctoral students around the world studying JNCL
Beyond Batten Disease Foundation and NCL-Stiftung host a symposium in Hamburg, Germany, that brings together doctoral students studying juvenile Batten disease from around the globe. January 26
The DNA sequencing carrier screening test developed
The DNA sequencing carrier screening test developed by BBDF and the National Center for Genome Research is launched as an in-house diagnostic tool. Further development of the test as a commercial product will provide royalties to the foundation to fund more juvenile Batten disease research. 
April 26
The Will Herndon Fund Raises over $1 Million
The Will Herndon Fund and their community in The Woodlands, Texas raises over $1 million for Batten disease research. August 26
BBDF and BDSRA design state of the art merit review process
BBDF, in partnership with Batten Disease Support and Research Association, designs a state of the art scientific merit review process that will serve as a guide to get research discoveries translated into treatment as quickly as possible. 
June 26
The foundation hosts Date Night with Keith Urban
The foundation hosts Date Night with Keith Urban, bringing international exposure and raising over $800,000. 
December 31
Carrier screening test makes cover of TIME magazine
January 26
Almost $1 million in grants announced to develop treatment and cure for Batten disease
Beyond Batten Disease Foundation and Batten Disease Support and Research Association (BDSRA), working together in close partnership with more than 20 other nonprofits around the world, announces grants totaling almost $1 million for research to develop treatments and a cure for Batten disease. February 26
BBDF announces collaboration to co-fund the creation of the Collaborative CNS Screening Initiative (CCSI)
Beyond Batten Disease Foundation, the Alzheimer’s Drug Discovery Foundation (ADDF), and the National Multiple Sclerosis Society announces a funding collaboration to support the creation of the Collaborative CNS Screening Initiative (CCSI), a central repository of chemical compounds that have shown significant Central Nervous System (CNS) activity. 
April 26
The foundation awards postdoctoral fellowship to Dr. Matthew Miscsenyi of Albert Einstein College of Medicine
Beyond Batten Disease Foundation awards a postdoctoral fellowship to Matthew Micsenyi, PhD of the Albert Einstein College of Medicine, New York. May 26
BBDF develops a milestone-driven timeline for clinical trials and FDA approval
Beyond Batten Disease Foundation develops a milestone-driven, "go/no-go" decision point timeline for clinical trials and FDA approval. 
June 26
BBDF co-supports to expansion of ADDF ACCESS
Beyond Batten Disease Foundation, together with Bright Focus, a foundation dedicated to the fight against brain disease and vision loss, supports the expansion of ADDF ACCESS. May 26
Preliminary data made possible by BBDF funding leads to additional grant support
Preliminary data made possible by BBDF funding leads to additional grant support from the Collaborative Research Fund of the Virginia and LE Simmons Family Foundation, two grants from the March of Dimes, and two grants from the National Institute of Neurological Disorders and Stroke (NINDS) of the National Institutes of Health totaling over $3.2 million. July 26
The foundation's scientific merit and review processes lead to almost $2.1 million in funding
The foundation’s scientific merit and translational review processes lead to almost $2 million in collaborative research funding from Ireland, Great Britain, Germany, Finland and the US. August 26
BBDF collaborates with 11 drug discovery centers around the world
The foundation begins collaborating with 11 drug discovery centers around the world to share promising compounds with other neurodegenerative diseases. By doing this together, we are saving millions of dollars and accelerating the screening process. In addition, we are leveraging funds with major partners like the Alzheimer’s Association and the MS Society. 
October 26
Charter members join the BBDF Development Board
127 charter members join the BBDF Development Board for a two year term. These active volunteers will serve as ambassadors to help the foundation grow and accomplish its mission by extending the reach of BBDF’s community, and raising awareness and funds for research to find a cure for juvenile Batten disease. 
November 26
Luke and Rachel Batten Foundation donates $40,000
BBDF collaborates with the Luke and Rachel Batten Foundation (LRBF) to provide funding to support the continuation of the promising research of Marco Sardiello, Ph.D., at the Jan and Dan Neurological Research Institute at Texas Children’s Hospital. LRBF and BBDF worked together to initiate and fund the first phrase of this project in 2012. 
January 26
The foundation co-funds expansion of European Batten patient registry, DEM Child
Beyond Batten Disease Foundation, together with Biomarin Pharmaceutical, LLC, Batten Disease Support and Research Association, and Noah’s Hope supports expansion of the European Commission-funded project DEM-CHILD (Germany, UK, Finland, Italy, India). February 26
Beyond Batten makes the Innovator Spotlight from FasterCures
Beyond Batten makes the Innovator Spotlight from FasterCures. FasterCures, a center of the Milken Institute, is an “action tank” that works to speed up and improve the medical research system. 
April 26
BBDF awards a $1.7 million grant to the Jan and Dan Duncan Neurological Research Institute
Beyond Batten Disease Foundation awards a $1.7 million grant to the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital in Houston, Texas to explore whether the pharmacological activation of TFEB can be used to activate the lysosomal system and to promote cellular clearance in JNCL, which in turn will ameliorate disease progression. May 26
BBDF funds the creation of the largest and first, genetically diverse, collection of induced pluripotent stem (iPS) cells in juvenile Batten disease
Beyond Batten Disease Foundation is funding the creation of the largest and first, genetically diverse, collection of induced pluripotent stem (iPS) cells in juvenile Batten disease. IPS cells are produced by artificially “turning back the clock” of skin cells to a time when they were stem cell-like and capable of becoming any cell in the body. With a pharmaceutical nudge, the embryonic cells will be pushed to become, not the skin cells they once were, but brain cells. November 7
BBDF selected for Innovator presentation at FasterCures Partnering for Cures
Beyond Batten Disease Foundation (BBDF) is selected for one of 30 Innovator presentations at the 6th annual FasterCures Partnering for Cures meeting held November 17, 2014 in New York City. Selected through a competitive proposal process, these partnerships are aimed at reducing the time and cost of getting new medical solutions from discovery to patients. December 26
BBDF partners with the New York Academy of Sciences to host
Beyond Batten Disease Foundation (BBDF), together with the New York Academy of Sciences (NYAS) hosted a “Drug Targeting the Lysosome Workshop” at the NYAS Conference Center at 7 World Trade Center. 
October 27
Youth Leadership Committee founded
Recognizing the young philanthropists in the community, BBDF founds the Youth Leadership Committee. December 26
BBDF partners with NCL-Stiftung to award $344,742 to research team of Dr. Marco Sardiello, Baylor College of Medicine, Houston
Beyond Batten Disease Foundation partners with NCL-Stiftung (Foundation) of Germany to award $344,742 to the research team of Dr. Marco Sardiello, Neurological Research Institute at Texas Children's Hospital and Baylor College of Medicine in Houston. December 26
BBDF co-sponsors the 14th International Conference on Neuronal Ceroid Lipofuscinoses (Batten Disease) & Patient Organisation meeting
Beyond Batten Disease Foundation, together with the NCL-Stiftung, BDSRA, Batten Disease Family Association of the UK and Bee for Batten, co-sponsors the 14th International Conference on Neuronal Ceroid Lipofuscinoses (Batten Disease) & Patient Organisation meeting. December 26
BBDF and Evotec sign agreement to validate emerging drug target and translate the findings across the
Beyond Batten Disease Foundation and Evotec AG have signed an agreement to validate emerging drug targets in juvenile Batten disease and translate promising findings across the drug discovery “Valley of Death” between fundamental discovery and applied medicine. January 26
BBDF selected as awardee for Platform for Engaging Everyone Responsibly
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. 
April 26
5th and Final Run to the Sun raises over $322,000
The Fifth and Final Run to the Sun brought the event's total fundraising over the past 5 years to $1.6 million. Over 320 runners and 250 volunteers stayed up overnight for the run from Enchanted Rock to Austin. May 26
BBDF and the New York Academy of Sciences release a new podcast.
Beyond Batten Disease Foundation and the New York Academy of Sciences release a new podcast: “Curing Human Diseases: Targeting the Lysosome” June 26
BBDF and Brain Canada announce a new partnership to promote large-scale, high-quality research in juvenile Batten disease across Canadian institutions
Beyond Batten Disease Foundation and Brain Canada announce a new partnership to promote large-scale, high-quality research in juvenile Batten disease across Canadian institutions. July 26
BBDF renowned Italian researcher publishes groundbreaking research in top international scientific publication
Published by BBDF-funded investigators Andrea Ballabio, Diego Medina and Carmine Settembre, investigators show that lysosomal calcium signaling regulates cellular recycling processes shown to be dysfunctional in juvenile Batten disease. August 26
BBDF delivers a grant to Cardiff University to Dr. Emyr Lloyd-Evans to explore a new hypothesis that has implications for juvenile Batten (CLN3)
Beyond Batten Disease Foundation delivers a grant to Cardiff University to Dr. Emyr Lloyd-Evans to explore a new hypothesis that has implications for juvenile Batten (CLN3). Based upon findings in genetically-engineered juvenile Batten disease mice, investigators hypothesize that children and young adults exhibit toxic levels of Ca,2+ suggesting that CLN3 protein plays a role in Ca2+ regulation. September 26
Beyond Batten Disease Foundation delivers a grant to Massachusetts General Hospital and Harvard Medical School funding Susan Cotman, PhD to determine the cellular location and function of the CLN3 protein
Beyond Batten Disease Foundation delivers a grant to Massachusetts General Hospital and Harvard Medical School funding Susan Cotman, PhD to determine the cellular location and function of the CLN3 protein. October 26
Craig Benson, co-founder of Beyond Batten Disease Foundation, is appointed to the Jan and Dan Duncan Neurological Research Institute (NRI) International Advisory Board at Texas Children’s Hospital in Houston
Craig Benson, co-founder of Beyond Batten Disease Foundation, is appointed to the Jan and Dan Duncan Neurological Research Institute (NRI) International Advisory Board at Texas Children’s Hospital in Houston. November 26
Joseph Glyksys, MD, PhD, publishes, “Diazepam effect during early neonatal development correlates with neuronal Cl”
Joseph Glyksys, MD, PhD, recipient of the American Brain Foundation/Beyond Batten Disease Foundation Clinical Research Training Fellowship in juvenile Batten disease publishes, “Diazepam effect during early neonatal development correlates with neuronal Cl” in the Annals of Clinical and Translational Neurology. These results lay the groundwork for Dr. Glykys’ work understanding why seizures occur in juvenile Batten disease. November 27
BBDF partners with Evotec to execute a 24-month plan
BBDF partners with Evotec to execute a 24-month plan to validate two lead compounds and to prepare an FDA Investigational New Drug application for approval to conduct clinical trials. To advance Beyond Batten Disease Foundation-funded discoveries beyond publication and into treatments, BBDF contracts with Evotec™, a broad range target-identification to preclinical neuroscience and metabolic disease company, for $2.2 million to put discoveries through their paces. November 28
BBDF co-sponsors the 13th Annual National NCL Congress
Beyond Batten Disease Foundation co-sponsors the 13th annual National NCL Congress hosted on November 2, 2015 in Hamburg, Germany. This year’s focus is on imaging techniques and calcium regulation in juvenile Batten disease-- two timely areas of investigation in our quest to learn more about the CLN3 protein and to identify biomarkers marking disease progression and eventually, response to treatment. December 31
To date, BBDF-funded research has resulted in 18 publications, cited 1,836 times
Over 40% of all peer-reviewed publications in 28,000 science and technology journals are never cited beyond a single publication. To date, funding from Beyond Batten Disease Foundation has resulted in 18 publication cited 1,835 times as the basis for or in support of additional investigations into juvenile Batten and other diseases in laboratories around the world. 
September 17
BBDF hosts first Family Night at Schlitterbahn Waterpark
BBDF hosts first Family Night: splashin' for a passion at Schlitterbahn Waterpark. With over 400 in attendance, families enjoyed private access to the park while benefitting BBDF. 
October 26
First Annual
Jessica Curran hosts the first annual "A Cure in the Nick of Time" Run/Walk in Duxbury, MA, in honor of her brother, Nick, who is diagnosed with juvenile Batten disease. The inaugural run raised over $25,000, showing the immense community support for the Curran Family. 
November 15
2014–2015 Annual Report
December 31
Youth Leadership Committee grows to over 60 Austin-area students
In its second year, the Youth Leadership Committee includes over 60 students from 9 high schools. January 26
Beyond Batten Disease Foundation grantee, Andrea Ballabio, MD, is awarded the 2016 Louis-Jeantet Prize for Medicine
Dr. Andrea Ballabio, MD, Beyond Batten Disease Foundation grantee at the Jan and Dan Duncan Neurological Research Institute (NRI) at Texas Children’s Hospital in Houston Texas and founder and director of the Telethon Institute of Genetics and Medicine (TIGEM) in Naples Italy, is awarded the 2016 Louis-Jeantet Prize for Medicine. Established in Geneva, Switzerland. Read more here. 
April 7
Hugs for Hudson and BBDF Join Forces
For Immediate Release Contact: Mary Beth Kiser - 512-275-2600 - mbkiser@beyondbatten.org Amy Phill... 
September 25
2016 Family Night at Schlitterbahn
In September 2016, over 550 supporters attended the 2nd Annual Family Night at Schlitterbahn Waterpark in New Braunfels. 
September 10
2016 Hope Under the Stars
The Will Herndon Fund's 8th Annual Hope Under the Stars grossed over $630,000 making it our most successful event ever! 
November 16
Austin BE First Party featured in Austin 360!
Beyond Batten. First, there’s the house. None other like it in Austin. Dimensional Fund head Da... 
November 16
BBDF Research Featured in Houston Chronicle
Six years ago, doctors told Missy and Wayne Herndon their 6-year-old son had a rare genetic disorder... November 16
Beyond Batten Disease Foundation, Noah’s Hope, Hope 4 Bridget and Batten Disease Research and Support Association Partner to Support the World’s Largest and most Comprehensive Batten Patient Registry
For Immediate Release Contact: Angela Hale Red Media Group Beyond Batten Disease Foundation, ... 
November 21
Introducing the BE Project
Beyond Batten Disease Foundation is proud to introduce the BE Project! The BE Project is a 24 month,... 
December 15
Believe! The 2015-2016 Annual Report
It's HERE! Click to see the 2015/2016 Annual Report.... 
November 30
Battling Batten Disease: Baton Rouge Parent's Magazine
Published November 30, 2016 By Madeline Rathle Christiane Benson, 14, loves snow skiing, walking... 
March 1
Garland Launches Be A Hero Campaign
Garland Benson launched a campaign to get 100,000 friends to donate $10 each in order to help fund a treatment for his sister. January 5
Beyond Batten Disease Foundation Announces New Funding for Metabolomics to Drive Biomarker Discovery in juvenile Batten disease
Beyond Batten Disease Foundation Announces New Funding for Metabolomics to Drive Biomarker Discovery... 
February 10
BBDF Funded Research Featured in Nature Communications
BBDF is very proud of the research we have funded over the last 8 years. We are especially honored... 
September 24
2017 Family Night at Schlitterbahn
The 3rd Annual Family Night at Schlitterbahn brought together family supporters splashin' for a passion and netted $83,452 for research! 
September 30
3rd Annual A Cure in the Nick of Time
The 3rd Annual A Cure in the Nick of Time Run/Walk, organized by Jessica Curran and her family, was held in September in Duxbury, Massachusetts! The event raised over $31,000 for research. 
October 3
Texas First Lady Cecilia Abbott Opens Her Home to BBDF
First Lady Cecilia Abbott opened her home to allow BBDF to share the message about Batten disease and the treatment in sight. 
October 14
2017 Hope Under the Stars
With nearly 900 guests in attendance, the Will Herndon Fund's 9th Annual Hope Under the Stars was an amazing event! 
October 23
New York Stem Cell Foundation and Beyond Batten Disease Foundation Announce the Availability of Stem Cell Lines for Research
The New York Stem Cell Foundation (NYSCF) and Beyond Batten Disease Foundation (BBDF) announce the availability of a unique and comprehensive collection of juvenile Batten (CLN3) disease induced pluripotent stem (iPS) cells. The two organizations partnered to develop this resource for scientists around the world. 
October 25
Beyond Batten Disease Foundation Annual Report 2016/2017
BBDF's 2016/2017 Annual Report is filled with research updates, inspiration, donor recognition and highlights. 
March 21
Batten Families Gather at TCH for Research Update
For the first time in foundation history, affected families from all over the world gathered at the ... 
May 9
FDA Meeting Update
In May BBDF had its first meeting with the FDA to get input and direction on our plan for clinical ... 
July 11
BBDF Engages Camargo Pharmaceutical Services for $1.6 Million
Beyond Batten Disease Foundation announces we have engaged Camargo Pharmaceutical Services, LLC (“Camargo”) for $1.6 million for regulatory consulting and strategic development of the 505(b)(2) regulatory pathway for the development of BBDF-101 for the treatment of juvenile Batten (CLN3) disease. 
August 13
To date, funding from Beyond Batten Disease Foundation has resulted in 19 publications cited over 2,000 times
Over 40% of all peer-reviewed publications in 28,000 science and technology journals are never cited beyond a single publication. To date, funding from Beyond Batten Disease Foundation has resulted in 19 publications cited over 2,000 times as the basis for- or in support of additional investigations into juvenile Batten and other diseases in laboratories around the world. 
September 24
BBDF 101 - Next Steps to Clinical Trials
In July, BBDF sponsored the Batten Disease Support and Research Association Family Conference where ... 
November 5
Researchers solve mystery surrounding form of Batten disease
Researchers from the Jan and Dan Duncan Neurological Research Institute at the Texas Children’s Hospital and Baylor College of Medicine have discovered... 
December 27
Garland Benson Makes National News
Garland Benson was featured on CBS Evening News and again Friday morning on We Are Austin. Garland’s sister, Christiane, suffers from Batten disease, and he has pledged to raise $1 million of the $6 million needed to advance a treatment to clinical trials. 
January 8
Garland Benson Reaches His $1M Goal
Impassioned by his sister's life-long struggle with Juvenile Batten disease, Garland Benson pledged to raise $1M of the $6M needed to advance a promising treatment to clinical trials. He did it! Click the link below to hear his heartfelt thank you. 
January 25
BE Project - Thank You from the Bensons
In 2016, after 8 years of funding research for Batten disease, our research team was ready to take the next steps to advance our discovery of a treatment to slow the progression of Batten disease. As you know, The Be Project was launched to raise $6 million in 24 months in order to validate their findings, create patient registries, and advance a host of other necessary steps to prepare for FDA engagement and clinical trials. And, we did just that. WE DID IT!!! We achieved our goal, and we hope you feel so proud and take ownership in this enormous accomplishment! 
February 14
Garland Benson Honored at AFP Greater Austin Chapter Philanthropy Day Luncheon
On Thursday, Feb. 14, Garland Benson was honored at the Association of fundraising Professionals Greater (AFP) Austin Chapter’s Philanthropy Day Luncheon, where he received the Kelly Davidson Memorial Outstanding Philanthropic Youth award. “We did it,” he announced to a ballroom pact with Austin’s most giving citizens. The crowd joyfully applauded Garland’s philanthropic work that ultimately raised over $1 Million to help advance a potentially life-saving treatment for his sister, Christiane, to clinical trials. We at Beyond Batten are so proud of Garland and grateful for his passion. Congratulations Garland! 
April 7
Battlin’ Batten in Baffin Fishing Tournament Exceeds $400,000 in Total Donations!
Friends and supporters of Beyond Batten Disease Foundation gathered on Sunday, April 7 for the 10th annual Battlin' Batten in Baffin Fishing Tournament in Baffin, TX. 
April 30
Rare Sisters Hosts First-Ever Expect Miracles 5K
The event raised nearly $40,000 to support juvenile Batten disease research, advancing us closer to a clinical trial and viable treatment. 
August 14
2nd Annual Bind Up Batten Golf Charity
Bind Up Batten brought together the community on July 20, 2019, for the 2nd Annual Batten Disease Go... 
March 18
Judy and Courtney Stecker establish the Charles
Judy and Courtney Stecker establish the Charles "Wheeler" Stecker Research Fund to join forces in raising funds for a cure after their son Wheeler was diagnosed with Batten Disease. 
April 30
Danielle Kerkovich is Recognized
BBDF's principal scientist, Danielle Kerkovich, recieves recognition for her paper as one of the most read in Molecular Genetics & Genomic Medicine. August 26
The FDA awarded Orphan Drug and Rare Pediatric Disease designations to Beyond Batten Disease Foundation for BBDF-101
The FDA has awarded Orphan Drug and Rare Pediatric Disease designations to Beyond Batten Disease Foundation for BBDF-101. 
November 27
Manuscript Accepted by Fundamental & Clinical Pharmacology
"NEUROPHARMA: Evaluating and modulating TFEB in the control of autophagy: towards new treatments in CNS disorders" manuscript by da Costa, Anaelle; Metais, Thibaud; Mouthon, Franck; Kerkovich, Danielle; Charvériat, Mathieu, was successfully accepted by the journal "Fundamental & Clinical Pharmacology".