Updates

September 25, 2020September 25, 2020
Early Spinal Cord Damage Found in Infantile Batten Disease Mouse Model

An examination of the spinal cords of mice that lack the PPT1 protein, the underlying cause of infantile Batten disease, revealed the loss of ... Read more
September 23, 2020September 21, 2020
Why the Fight Against Rare Disease Is Like High-stakes Soccer

We all wish we could relive experiences from our childhood — those fleeting moments that turn into minutes and hours and days faster than ... Read more
September 18, 2020September 18, 2020
New Approach to Correct CLN3 Mutation Shows Promise in Juvenile Batten Disease Mouse Model

Treating a juvenile Batten disease mouse model with antisense oligonucleotides to correct its underlying disease-causing mutation led to less waste buildup in the brain, improved ... Read more
September 11, 2020September 11, 2020
Abeona Partners With Taysha to Advance ABO-202 as Potential Gene Therapy

Abeona Therapeutics and Taysha Gene Therapies have entered into agreements concerning ABO-202, an investigative gene therapy for infantile Batten disease. The agreements give Taysha ... Read more
September 4, 2020September 4, 2020
Same But Different Photo Contest Celebrates People With Rare Diseases

Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography competition to raise awareness for ... Read more

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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

Updates

Early Spinal Cord Damage Found in Infantile Batten Disease Mouse Model

Why the Fight Against Rare Disease Is Like High-stakes Soccer

New Approach to Correct CLN3 Mutation Shows Promise in Juvenile Batten Disease Mouse Model

Abeona Partners With Taysha to Advance ABO-202 as Potential Gene Therapy

Same But Different Photo Contest Celebrates People With Rare Diseases

Subscribe to our mailing list

Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input

FDA Names Investigational Therapy BBDF-101 Orphan Drug, Rare Pediatric Disease

Good News for CLN1 Disease and a Sprint to the Finish

EveryLife Foundation Launches Scholarship Fund for Rare Disease Community in US

Neurogene’s CLN7 Gene Therapy Granted FDA Orphan Drug Designation

When Is It OK to Quit Fighting Rare Disease?

New Streaming Channel Showcases Rare Disease Films

Case Report Describes Rare Case of Newborn Diagnosed With CLN2 Disease

URMC Hopes New IDDRC Research Designation Leads to Scientific Advances

Your Rare Disease Reading List for Quarantine: Part 2

Role of CLN6 in Batten Discovered, Shedding Light on Disease

Your Rare Disease Reading List for Quarantine: Part 1

Lymphocyte Vacuolization Might Be Used to Measure Batten Severity

Neurogene’s Gene Therapy for CLN5 Named Orphan Drug by FDA

CLN2 Mouse Model Shows Small Vesicles May Help Deliver TPP1 to Brain

Subtle Signs of Brain Atrophy Should Prompt Testing, Study Says

Coalition Will Address Racial Disparities in Rare Disease Communities

Pakistani Study Describes New Mutations That Cause CLN5 Disease

Common Batten Tests Spot Biomarkers in Mouse Model That Track Progression

COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

Study Examines Reduction in CLN6’s Ability to Prevent Protein Aggregation

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

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Early Spinal Cord Damage Found in Infantile Batten Disease Mouse Model

Why the Fight Against Rare Disease Is Like High-stakes Soccer

New Approach to Correct CLN3 Mutation Shows Promise in Juvenile Batten Disease Mouse Model

Abeona Partners With Taysha to Advance ABO-202 as Potential Gene Therapy

Same But Different Photo Contest Celebrates People With Rare Diseases

Subscribe to our mailing list

Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input

FDA Names Investigational Therapy BBDF-101 Orphan Drug, Rare Pediatric Disease

Good News for CLN1 Disease and a Sprint to the Finish

EveryLife Foundation Launches Scholarship Fund for Rare Disease Community in US

Neurogene’s CLN7 Gene Therapy Granted FDA Orphan Drug Designation

When Is It OK to Quit Fighting Rare Disease?

New Streaming Channel Showcases Rare Disease Films

Case Report Describes Rare Case of Newborn Diagnosed With CLN2 Disease

URMC Hopes New IDDRC Research Designation Leads to Scientific Advances

Your Rare Disease Reading List for Quarantine: Part 2

Role of CLN6 in Batten Discovered, Shedding Light on Disease

Your Rare Disease Reading List for Quarantine: Part 1

Lymphocyte Vacuolization Might Be Used to Measure Batten Severity

Neurogene’s Gene Therapy for CLN5 Named Orphan Drug by FDA

National Survey Seeks to Assess Full Social, Economic Burdens of Rare Diseases in US

CLN2 Mouse Model Shows Small Vesicles May Help Deliver TPP1 to Brain

What Can COVID-19 Teach Us About Batten Disease?

Subtle Signs of Brain Atrophy Should Prompt Testing, Study Says

Coalition Will Address Racial Disparities in Rare Disease Communities

Pakistani Study Describes New Mutations That Cause CLN5 Disease

Common Batten Tests Spot Biomarkers in Mouse Model That Track Progression

COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

Study Examines Reduction in CLN6’s Ability to Prevent Protein Aggregation

Video Games Connect Chronically Ill Children Isolated at Home, Hospital

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