Updates

February 22, 2021February 22, 2021
Rare Disease Day at NIH, Set for March 1, Growing Year by Year

Rare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place on March 1, will feature panel discussions, patient stories, ... Read more
February 19, 2021February 19, 2021
Eurordis Survey: Healthcare Experience Worse for Rare Disease Patients

People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of an international survey conducted ... Read more
February 19, 2021February 19, 2021
CLN3 Gene Therapy Showing Safety, Possible Efficacy in Trial, Amicus Reports

AT-GTX-502, Amicus Therapeutics’ investigational gene therapy for juvenile Batten disease, has been well-tolerated and is showing a potential to stabilize early signs of the disease ... Read more
February 17, 2021February 12, 2021
My Sister Is Gone, but She’s Still Teaching Me to Live in the Moment

Batten disease took my sister from me, but it didn’t take her love for life. Now, more than two years after her untimely death ... Read more
February 12, 2021February 12, 2021
Gene Therapy Directly to Brain Helps Preserve Vision in CLN6 Mouse Model

A single injection into the brain of an investigational gene therapy now in clinical trials partly prevented retinal degeneration and vision loss in a ... 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

Rare Disease Day at NIH, Set for March 1, Growing Year by Year

Eurordis Survey: Healthcare Experience Worse for Rare Disease Patients

CLN3 Gene Therapy Showing Safety, Possible Efficacy in Trial, Amicus Reports

My Sister Is Gone, but She’s Still Teaching Me to Live in the Moment

Gene Therapy Directly to Brain Helps Preserve Vision in CLN6 Mouse Model

Subscribe to our mailing list

Pandemic Won’t Stop Rare Disease Day on Feb. 28

Analysis of Proteins in Urine Identifies Potential Disease Biomarkers

NORD’s 6th ‘State Report Card’ Notes Progress, Raises Concerns

FDA Issues Guidance on Developing Rare Disease Treatments

COVID-19 Vaccines Pose Little Risk to Rare Disease Patients, FDA, CDC Say

Vision Loss More Severe in Juvenile Batten Than in Stargardt, Study Says

NORD’s Caregiver Respite Program Continues Through Pandemic

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

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

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

Rare Disease Day at NIH, Set for March 1, Growing Year by Year

Eurordis Survey: Healthcare Experience Worse for Rare Disease Patients

CLN3 Gene Therapy Showing Safety, Possible Efficacy in Trial, Amicus Reports

My Sister Is Gone, but She’s Still Teaching Me to Live in the Moment

Gene Therapy Directly to Brain Helps Preserve Vision in CLN6 Mouse Model

Subscribe to our mailing list

Pandemic Won’t Stop Rare Disease Day on Feb. 28

Analysis of Proteins in Urine Identifies Potential Disease Biomarkers

NORD’s 6th ‘State Report Card’ Notes Progress, Raises Concerns

FDA Issues Guidance on Developing Rare Disease Treatments

COVID-19 Vaccines Pose Little Risk to Rare Disease Patients, FDA, CDC Say

Vision Loss More Severe in Juvenile Batten Than in Stargardt, Study Says

NORD’s Caregiver Respite Program Continues Through Pandemic

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

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