Updates

November 26, 2020November 26, 2020
Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe

The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their ... Read more
November 20, 2020November 20, 2020
Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry

Leaders in the U.S. rare disease community came together recently for a webinar to present helpful information on how to start a nonprofit and ... Read more
November 20, 2020November 20, 2020
BDFA Seeks Funds for Potential Brineura Study to Try Saving Children’s Sight

The Batten Disease Family Association (BDFA) is moving forward with a fundraising campaign aimed at developing a United Kingdom study that would use Brineura ... Read more
November 18, 2020November 17, 2020
Giving Thanks, Despite Batten Disease

Thanksgiving began as a simple gathering hundreds of years before the invention of football and shopping malls. For the Pilgrims, this special day was ... Read more
November 13, 2020November 13, 2020
Taysha, Invitae Partner on Rapid Genetic Testing for Lysosomal, Epileptic Disorders

Taysha Gene Therapies has announced a partnership with Invitae to support two of Invitae’s programs that enable more rapid diagnoses for patients with lysosomal ... 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

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

Subscribe to our mailing list

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

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

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

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

Subscribe to our mailing list

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

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