Updates

January 18, 2019January 18, 2019
CLN3 Disease Should be Considered When Diagnosing AVM, Researchers Say

A team of Italian researchers suggests that CLN3 disease, also known as juvenile Batten disease, should be included in the differential diagnosis of people ... Read more
January 11, 2019January 11, 2019
Both Rare and Classic Juvenile Batten Types Show Changes in Skeletal Muscle Cells, Study Finds

The activation of a recycling pathway, a process called autophagy, which leads to large storage vesicles in muscle cells, is a feature of all ... Read more
January 4, 2019January 4, 2019
Amicus Therapeutics Recruiting Children with CLN3 Batten Disease for Gene Therapy Trial

Amicus Therapeutics is recruiting children for its new Phase 1/2 clinical trial that will assess the safety and efficacy of an investigational gene therapy ... Read more
December 20, 2018December 20, 2018
Rising Healthcare Costs Strain EU Budgets Even as New Therapies Flourish

Cash-strapped governments across the 28-member European Union are struggling to control runaway healthcare expenditures — at exactly the same time as the promise of ... Read more
December 18, 2018December 18, 2018
New Mutations Linked to Infantile and Late-Infantile Batten Disease Identified in Study of Indian Patients

New mutations have been found in PPT1 and TPP1, the two genes associated with neuronal ceroid lipofuscinoses type 1 and type 2 (NCL1 and ... Read more

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News

CLN8 Deficiency Disrupts Cell-Signaling Pathways, Study Reports
December 4, 2018December 4, 2018
A type of Batten disease, called CLN8 disease, is associated with the failure of ...
News

Mutations in CLN8 Block the Transport of Lysosomal Enzymes, Leading to Batten Disease, Study Shows
November 27, 2018November 27, 2018
Genetic mutations in the CLN8 gene block the transport of lysosomal enzymes to lysosomes, the cellular ...
News

Regenxbio’s One-time Gene Therapy RGX-181 Wins FDA’s Orphan Drug Designation
November 20, 2018November 20, 2018
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Regenxbio’s one-time gene therapy RGX-181 for ...
News

Abeona Acquires Rights to Develop Gene Therapies Using NAV AAV9 Gene Delivery System
November 13, 2018November 13, 2018
Abeona Therapeutics has acquired the global rights to REGENXBIO’s proprietary NAV AAV9 delivery system to develop ...
News

Canine Models of Batten Disease May Help to Develop New Therapeutic Approaches, Review Says
November 6, 2018November 6, 2018
Naturally occurring canine models of several types of neuronal ceroid lipofuscinoses (NCLs), also known as ...
News

#NORDSummit – Major Issues on Table for Rare Disease Patients in US as Midterm Elections Approach
October 30, 2018October 29, 2018
With the U.S. midterm elections now less than two weeks away, patient advocacy groups ...

CLN3 Deletion Affects Genes Responsible for Production of Type of Fat in Brain, Mouse Study Finds
October 30, 2018October 30, 2018
A genetic deletion in the CLN3 gene impairs the normal function of genes involved in the ...
Researchers Dissect Mechanisms Involved in CLN5 Release Using Amoeba Model
October 26, 2018October 26, 2018
Using a species of amoeba as a model organism, researchers have found chemical modifications ...
Researchers ID 8 Potential Biomarkers of Disease Severity in Late Infantile Batten Disease
October 23, 2018October 23, 2018
Compounds Similar to Flupirtine Seem Promising for the Treatment of Batten Disease, Study Reports
October 16, 2018October 16, 2018
CLN3 Protein Involved in Maintaining Water Balance, Batten Disease Model Shows
October 12, 2018October 12, 2018
Advocacy Groups, Doctors Question Rising Prices of Rare Disease Treatments
October 9, 2018October 9, 2018

Researchers Explore Caspase-1 as Potential Therapeutic Target for CLN3 Disease
October 2, 2018October 2, 2018
Caspase-1 — an enzyme involved in several cellular processes, including inflammatory responses — is ...
Amicus Therapeutics’ Expanded Portfolio Includes Potential Gene Therapies for Batten Disease
September 28, 2018September 28, 2018
Exome Sequencing is a Valuable Tool for Diagnosis of Pediatric Batten Disease
September 25, 2018September 25, 2018
Search for Biomarkers in CLN3 Mouse Model Fails to Yield Results, Study Reports
September 21, 2018September 21, 2018

#NORDSummit – More Than 700 Expected to Attend Oct. 15-16 Rare Disease Summit in Washington
September 21, 2018
More than 700 medical experts, pharmaceutical executives, patient advocates, and others are expected to ...
Researchers Use CT Scans to Monitor Long-term Brain Alterations in Sheep Models of Batten Disease
September 18, 2018September 18, 2018
LivaNova Launches Study to Assess VNS Therapy in Drug-resistant Epilepsy Patients
September 11, 2018September 11, 2018

News

Regenxbio Moves Toward Human Clinical Trial for Investigational Gene Therapy RGX-181
September 4, 2018September 4, 2018
Regenxbio is developing a novel, one-time gene therapy, called RGX-181, for the treatment of ...
News

Batten Disease Biomarker Research Influenced by Husbandry Conditions Among Mice, Study Finds
August 31, 2018August 31, 2018
Husbandry conditions of mouse models of CLN3-Batten disease may influence research outcomes, such as ...
News

Nerve Cells’ Dysfunction May Contribute to Neurodegeneration in Several Types of Batten’s, Mouse Study Reveals
August 28, 2018August 28, 2018
Two types of nerve cells — microglia and astrocytes — are dysfunctional and may ...
News

FDA Accepts New Drug Application for Nayzilam Nasal Spray for Acute Treatment of Seizures
August 22, 2018August 22, 2018
The U.S. Food and Drug Administration has accepted a new drug application (NDA) for UCB’s ...
News

Motor Events in Juvenile Batten Disease Like Those Seen in Brain Injuries, Study Suggests
August 14, 2018August 14, 2018
Agitation and involuntary motor activity, which normally occurs in individuals who suffered traumatic brain ...
News

Healx Raises $10M to Speed Therapy Discovery for Batten, Other Rare Diseases
August 7, 2018August 7, 2018
Healx has raised $10 million to accelerate therapy discovery for rare diseases, including Batten disease. The company uses ...

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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