Updates

October 16, 2018October 16, 2018
Compounds Similar to Flupirtine Seem Promising for the Treatment of Batten Disease, Study Reports

Chemical compounds similar to flupirtine can effectively prevent cell death in Batten disease patient-derived cell lines, a study reports. These findings provide the first preclinical ... Read more
October 12, 2018October 12, 2018
CLN3 Protein Involved in Maintaining Water Balance, Batten Disease Model Shows

CLN3, the protein lacking in the juvenile form of Batten disease (CLN3 disease), is involved in maintaining water balance, which is key for cell survival and growth, ... Read more
October 9, 2018October 9, 2018
Advocacy Groups, Doctors Question Rising Prices of Rare Disease Treatments

Half a year has gone by since disgraced pharma executive Martin Shkreli was sentenced to seven years in federal prison for securities and wire ... Read more
October 2, 2018October 2, 2018
Researchers Explore Caspase-1 as Potential Therapeutic Target for CLN3 Disease

Caspase-1 — an enzyme involved in several cellular processes, including inflammatory responses — is a potential therapeutic target to revert some of the features ... Read more
September 28, 2018September 28, 2018
Amicus Therapeutics’ Expanded Portfolio Includes Potential Gene Therapies for Batten Disease

Amicus Therapeutics has gained global development and commercial rights to 10 gene therapy programs for lysosomal storage disorders, including three potential first-to-market therapies for Batten disease. ... Read more

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News

Exome Sequencing is a Valuable Tool for Diagnosis of Pediatric Batten Disease
September 25, 2018September 25, 2018
Exome sequencing, a technique that looks for gene mutations, is a valuable tool to ...
News

Search for Biomarkers in CLN3 Mouse Model Fails to Yield Results, Study Reports
September 21, 2018September 21, 2018
An animal study using a mouse model of CLN3 disease, the juvenile form of ...
News

#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 ...
News

Researchers Use CT Scans to Monitor Long-term Brain Alterations in Sheep Models of Batten Disease
September 18, 2018September 18, 2018
New Zealand researchers used computed tomography (CT) to measure and monitor brain alterations in ...
News

LivaNova Launches Study to Assess VNS Therapy in Drug-resistant Epilepsy Patients
September 11, 2018September 11, 2018
A new system of vagus nerve stimulation (VNS) therapy was implanted in the first patient with ...
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 ...

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 ...
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 ...
FDA Accepts New Drug Application for Nayzilam Nasal Spray for Acute Treatment of Seizures
August 22, 2018August 22, 2018
Motor Events in Juvenile Batten Disease Like Those Seen in Brain Injuries, Study Suggests
August 14, 2018August 14, 2018
Healx Raises $10M to Speed Therapy Discovery for Batten, Other Rare Diseases
August 7, 2018August 7, 2018
Experts Create New Guidelines for Optimal CLN2 Disease Management
August 3, 2018August 3, 2018

Discovery of Natural CLN7 Model in Type of Monkey May Help Advance Understanding of Disease
July 31, 2018July 31, 2018
Researchers have found a natural model of CLN7 disease, a rare form of Batten ...
‘Behind These Hands,’ a Novel-in-Verse, Illustrates Affects of Batten Disease Diagnosis
July 24, 2018July 24, 2018
New Insight into CLN5 Protein May Help Develop New Therapeutic Strategies, Study Reports
July 13, 2018July 13, 2018
Common Disease Mechanisms Found Between Type of Batten Disease and Form of Adult Dementia
July 10, 2018July 10, 2018

Progress in Gene and Cell Therapies Reason for Hope Among Batten Patients, Review Study Says
July 6, 2018July 6, 2018
Investigational therapies now in development — especially gene and cell therapies — are reason ...
Scientists Find Previously Unknown Role for Protein Linked to Juvenile Batten Disease
July 3, 2018July 3, 2018
Fruit Fly Research Examines Two Genes Involved in Batten Disease
June 29, 2018June 29, 2018

News

CellCept Safe, But Ineffective, in Phase 2 Study for CLN3 Disease
June 27, 2018June 27, 2018
Roche’s CellCept was safe, but lacked clinical benefits, during short-term use by patients with CLN3 ...
News

Newborn Screening Test Shows Promise for Detecting Late Infantile Batten Disease, Study Shows
June 22, 2018June 22, 2018
Researchers have developed a new newborn screening test that uses dried blood spots to more ...
News

Researchers Identify 5 New Mutations Linked to Form of Batten Disease
June 19, 2018June 19, 2018
Researchers have identified five new mutations in the CLN8 gene — the genetic factor ...
News

New Mouse Model of Late Infantile Batten Disease May Help Assess Potential Therapies
June 15, 2018June 15, 2018
A new mouse model of late infantile Batten disease may help to efficiently investigate ...
News

First Batten Disease Awareness Day a Milestone to Unite Global Community, Increase Knowledge
June 12, 2018June 12, 2018
The inaugural International Batten Disease Awareness Day was launched on June 9 under the theme “Come ...
Just Published, News

Neuron-Like Cells Derived from Patients May Offer Way to Better Understand and Treat Disease, Study Reports
June 8, 2018June 8, 2018
Neuron-like cells derived from a Batten patient’s skin cells may be a suitable way of ...

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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