Updates

January 8, 2021January 8, 2021
NORD Seeks Speakers for 2021 Virtual ‘Living Rare, Living Stronger’ Forum

The National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life experiences with rare diseases to speak at its upcoming virtual ... Read more
January 8, 2021January 8, 2021
Taysha, Catalent Partner to Advance Gene Therapies for Batten, Other Disorders

Taysha Gene Therapies announced that it has joined with Catalent, a pharmaceutical services provider, to support production of its potential gene therapies for disorders that ... Read more
December 18, 2020December 18, 2020
Cannabis Oil as Add-on Therapy Seen to Ease Severe Seizures in CLN6 Batten

Adding cannabis oil to standard anti-seizure medications may be an effective way of managing treatment-resistant status epilepticus — life-threatening seizures that require urgent medical ... Read more
December 16, 2020December 14, 2020
Walking the Path From Here to There

This will be my last post of 2020. But instead of wailing that the new year can’t possibly get here soon enough, I want ... Read more
December 11, 2020December 11, 2020
Enrollment Begins in Natural History Study of 2 Batten Disease Subtypes

Neurogene has enrolled the first patient in a natural history study of CLN5 and CLN7 disease, two forms of late-infantile Batten disease, the company announced. ... 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

NORD Seeks Speakers for 2021 Virtual ‘Living Rare, Living Stronger’ Forum

Taysha, Catalent Partner to Advance Gene Therapies for Batten, Other Disorders

Cannabis Oil as Add-on Therapy Seen to Ease Severe Seizures in CLN6 Batten

Walking the Path From Here to There

Enrollment Begins in Natural History Study of 2 Batten Disease Subtypes

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NORD Push for Rare Disease Advisory Councils Focus of Dec. 16 Public Meeting

Juvenile Batten Disease Treatment BBDF-101 Earns FDA’s OK for Pre-Clinical Plan

Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe

Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry

BDFA Seeks Funds for Potential Brineura Study to Try Saving Children’s Sight

Giving Thanks, Despite Batten Disease

Taysha, Invitae Partner on Rapid Genetic Testing for Lysosomal, Epileptic Disorders

NORD Hosting ‘RareLaunch’ Workshops to Help Start Rare Disease Non-profits

Amicus Gene Therapy Effective After 2 Years in CLN6 Batten Children, Interim Results Show

UK Organization ‘Same But Different’ Announces ‘Glimmer of Hope’ Photo Contest Winner

Brineura Now Available Through NHS Scotland to Children With CLN2 Disease

Telehealth ‘Helpful’ Alternative to In-person Care, Rare Disease Patients Say

CBD Treatment Found Effective for Severe Seizures in Batten Disease Case

Same But Different Asks Public to Vote on ‘Glimmer of Hope’ Calendar Photos

First Report Published of Batten and Moyamoya Diseases Occurring in Same Patient

Amicus’ Gene Therapy for CLN6 Batten Given PRIME Status in Europe

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

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

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

NORD Seeks Speakers for 2021 Virtual ‘Living Rare, Living Stronger’ Forum

Taysha, Catalent Partner to Advance Gene Therapies for Batten, Other Disorders

Cannabis Oil as Add-on Therapy Seen to Ease Severe Seizures in CLN6 Batten

Walking the Path From Here to There

Enrollment Begins in Natural History Study of 2 Batten Disease Subtypes

Subscribe to our mailing list

NORD Push for Rare Disease Advisory Councils Focus of Dec. 16 Public Meeting

Juvenile Batten Disease Treatment BBDF-101 Earns FDA’s OK for Pre-Clinical Plan

Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe

Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry

BDFA Seeks Funds for Potential Brineura Study to Try Saving Children’s Sight

Giving Thanks, Despite Batten Disease

Taysha, Invitae Partner on Rapid Genetic Testing for Lysosomal, Epileptic Disorders

NORD Hosting ‘RareLaunch’ Workshops to Help Start Rare Disease Non-profits

Amicus Gene Therapy Effective After 2 Years in CLN6 Batten Children, Interim Results Show

UK Organization ‘Same But Different’ Announces ‘Glimmer of Hope’ Photo Contest Winner

Batten Disease Changed Our Halloweens Long Before COVID-19

Brineura Now Available Through NHS Scotland to Children With CLN2 Disease

Telehealth ‘Helpful’ Alternative to In-person Care, Rare Disease Patients Say

CBD Treatment Found Effective for Severe Seizures in Batten Disease Case

Same But Different Asks Public to Vote on ‘Glimmer of Hope’ Calendar Photos

First Report Published of Batten and Moyamoya Diseases Occurring in Same Patient

Amicus’ Gene Therapy for CLN6 Batten Given PRIME Status in Europe

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

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