Updates

August 14, 2020August 14, 2020
New Streaming Channel Showcases Rare Disease Films

When the COVID-19 pandemic forced the postponement of a rare disease film festival originally slated for May, its organizers set out to find a ... Read more
August 7, 2020August 7, 2020
Case Report Describes Rare Case of Newborn Diagnosed With CLN2 Disease

A case report has described a rare case of a newborn diagnosed with CLN2 disease, or late infantile Batten disease, after a genetic analysis confirmed a ... Read more
July 31, 2020July 31, 2020
URMC Hopes New IDDRC Research Designation Leads to Scientific Advances

The University of Rochester Medical Center (URMC) hopes to parlay its new designation as an Intellectual & Developmental Disabilities Research Center (IDDRC) into scientific ... Read more
July 29, 2020July 24, 2020
Your Rare Disease Reading List for Quarantine: Part 2

Part two of a series. Read part one. If you’re like much of the world, you’ve spent more time at home than usual in ... Read more
July 24, 2020July 24, 2020
Role of CLN6 in Batten Discovered, Shedding Light on Disease

The proteins CLN6 and CLN8 — which are deficient in people with two forms of Batten disease — were found to interact directly with ... 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

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

Subscribe to our mailing list

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

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

Batten Disease: Life, Interrupted

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

Amicus’ CLN3 Batten Disease Gene Therapy Gets FDA Fast Track Designation

Rare Diseases Clinical Research Network Opens Online Survey on COVID-19

NORD Webinar Outlines COVID-19 Response on Financial, Policy Fronts

Industry leaders seek to advance innovative resources for rare and orphan disease communities amid COVID-19 outbreak

Brineura Shows Promise Delaying Disease Progression in Children with CLN2, Review Suggests

Surge in Telemedicine One ‘Good’ Outcome from COVID-19 Crisis, Doctors Say

NORD Opens COVID-19 Financial Aid Program for Rare Disease Community

Rare Disease Therapy Development: Finding a Way Forward

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

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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

Subscribe to our mailing list

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

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

Batten Disease: Life, Interrupted

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

Amicus’ CLN3 Batten Disease Gene Therapy Gets FDA Fast Track Designation

Rare Diseases Clinical Research Network Opens Online Survey on COVID-19

NORD Webinar Outlines COVID-19 Response on Financial, Policy Fronts

Industry leaders seek to advance innovative resources for rare and orphan disease communities amid COVID-19 outbreak

Brineura Shows Promise Delaying Disease Progression in Children with CLN2, Review Suggests

Surge in Telemedicine One ‘Good’ Outcome from COVID-19 Crisis, Doctors Say

NORD Opens COVID-19 Financial Aid Program for Rare Disease Community

Rare Disease Therapy Development: Finding a Way Forward

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