Updates

February 13, 2020February 13, 2020
Cases of Late Infantile Batten Disease and Their Genetic Mutations Discovered by Researchers in Saudi Arabia

Two new mutations in the DNA sequences of children with late infantile Batten disease, a type of neuronal ceroid lipofuscinoses (NCL), have been found ... Read more
February 10, 2020February 10, 2020
FDA Efforts to Bring Patients into Treatment Decisions Focus of NORD Webinar

The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 people and had a budget of $5.7 billion in 2019. ... Read more
February 6, 2020February 6, 2020
With Post-Brexit Move to Amsterdam Complete, EMA Renews Rare Disease Focus

The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business ... Read more
February 6, 2020February 6, 2020
Researchers Discover New Gene Variant Associated with Adult-onset Batten Initially Missed by Genetic Tests

A previously unidentified variant of a gene associated with adult-onset Batten disease (ANCL) was found in an affected family after initial genetic tests failed, ... Read more
January 30, 2020January 30, 2020
Enzyme’s Stability in Cells Should Be Considered When Developing New Replacement Therapies, Study Suggests

The stability of lysosomal proteins when tested in a laboratory setting is not equivalent to that observed in live cells, a study has found. ... Read more

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News

Juvenile Batten Disease Should Be Considered in Children With Rapid Vision Loss, Study Suggests
January 23, 2020January 23, 2020
Children with rapid and progressive vision loss — regardless of cognitive or behavioral problems ...
Columns, The Long Sprint - a Column by Laura King Edwards

Running to the Finish Line, for Taylor’s Sake
January 22, 2020January 21, 2020
I miss my sister. Many people in the rare disease community are surprised, even ...
News

Companies Unite to Support Free Gene Testing in Young Children with Seizures
January 16, 2020January 16, 2020
Neurogene, which is developing therapies to treat people with Batten disease, has joined with ...
News

Theranexus and BBDF Announce Agreement for Juvenile Batten Disease Treatment Trial
January 9, 2020January 9, 2020
Biopharmaceutical company Theranexus has reached an exclusive $20 million global agreement with Beyond Batten ...
News

New Insights into Batten Disease Help Inform Treatments Under Development, Review Says
January 2, 2020January 2, 2020
New research has deepened the understanding of the underlying causes of Batten disease, including ...
News

Fusion Protein Strategy Creates Trojan Horse for Future Batten Disease Treatment
December 19, 2019December 19, 2019
Those afflicted with lysosomal storage disorders such as Batten disease may benefit from a clever ...

Drinking Acidified Water May Have Beneficial Effects in Children With CLN3, Mouse Study Suggests
December 12, 2019December 12, 2019
Drinking acidified water may have beneficial effects for children with CLN3 disease, the juvenile ...
Gene Therapy Takes Center Stage at 2019 NORD Summit
December 10, 2019December 10, 2019
With so much recent publicity surrounding gene therapy, it’s no surprise that the topic ...
HHS Secretary Alex Azar Touts White House Efforts to Cure Rare Diseases
December 6, 2019
Genetic Test Can Provide Definitive Diagnosis of Batten Disease, Canadian Study Suggests
December 5, 2019December 5, 2019
CLN7 Protein is Involved in Communication Between Nerve and Muscle Cells, Batten Disease Model Shows
November 21, 2019November 21, 2019
Rare Disease Film Festival Highlights Patient and Researcher Unity
November 14, 2019

Fat Molecule May Help Fight CLN3 Disease, Mouse Study Suggests
November 14, 2019November 14, 2019
Treatment with galactosylceramide (GalCer) — a type of fat molecule — improved motor function, ...
Scientists Identify Proteins That May Become Biomarkers of Batten Disease
November 7, 2019November 7, 2019
Advancing Clinical Care Recommendations for CLN1 Disease
November 6, 2019November 1, 2019
Complex Fat Molecule Seen to Aid Lifespan, Ease Neuron Loss in Juvenile Batten Animal Study
October 31, 2019October 31, 2019

Single Gene Therapy Injection May Stabilize CLN6 Batten Disease in Children, Early Phase 1/2 Data Show
October 24, 2019October 24, 2019
Amicus Therapeutics‘ investigational gene therapy for CLN6 Batten disease may halt disease progression in ...
Gene Therapy Prolongs Lifespan, Alleviates Symptoms of Mice With CLN6 Disease, Study Shows
October 18, 2019October 18, 2019
Customized Oligonucleotides Treat Seizures in Child with CLN7 Disease in Record Time
October 11, 2019October 11, 2019

News

UFC Cheers Young Fan with Batten While Helping to Raise Disease Awareness
October 4, 2019October 4, 2019
The Ultimate Fighting Championship (UFC) lifted the spirits of a boy named Sebastian recently ...
Columns, The Long Sprint - a Column by Laura King Edwards

9 Things We Should Know About the Race to New Treatments
October 2, 2019September 30, 2019
I favor running analogies — even the name of this column pays tribute to ...
News

NORD 2019 Rare Disease Summit Set for Oct. 21-22 in Washington, DC
September 27, 2019September 26, 2019
Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, ...
News

UK Batten Families Welcome Brineura Access for CLN2 Patients Through NHS
September 20, 2019September 20, 2019
When Ollie Carroll was 5 years old, the British boy made history by becoming ...
News

Taylor’s Tale Co-founder Races to ‘Change the World’ for Batten Disease Community
September 6, 2019September 9, 2019
Laura King Edwards ran a half-marathon as a fifth anniversary nod to her sister’s first ...
News

Extracellular Vesicles May Enhance Delivery of TPP1 to the Brain for Replacement Therapy in CLN2, Early Study Suggests
August 30, 2019August 30, 2019
Extracellular vesicles derived from a type of immune cell called macrophage may hold potential ...

July 20, 2017July 20, 2017

North Carolina’s Indo Jax Surf Charities Helps Kids With Batten Disease, Other Special Needs

News

A small North Carolina nonprofit organization, Indo Jax Surf Charities, has recently gained attention for its set of free programs to help children with Batten disease and other special needs build self-esteem. Since launching these programs, Indo Jax has been featured ... Read more

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