Updates

May 25, 2020May 25, 2020
Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

European authorities must step up efforts to screen babies for a multitude of genetic disorders, a panel of experts suggested during a May 14-15 ... Read more
May 22, 2020May 22, 2020
Study Examines Reduction in CLN6’s Ability to Prevent Protein Aggregation

A reduction in the ability of the CLN6 protein to prevent other proteins from clumping together and forming aggregates can account at least partially ... Read more
May 21, 2020May 21, 2020
Video Games Connect Chronically Ill Children Isolated at Home, Hospital

Dara Riva always had a rule that her 10-year-old son could play video games only once a week. But then the COVID-19 pandemic struck, ... Read more
May 20, 2020May 20, 2020
Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first all-virtual conference, bringing some 1,500 delegates from 57 countries together ... Read more
May 20, 2020May 18, 2020
Batten Disease: Life, Interrupted

The message landed in my inbox just before lunch on Day 61 of the quarantine. I was working from home while making lunch 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

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

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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

Axovant, Invitae Offering Free Genetic Testing, Counseling for Lysosomal Storage Diseases

FDA Favors Clinical Trial of Potential Juvenile Batten Treatment, PLX-200

NORD’s Advice on COVID-19: You’re Stronger Than You Think

NORD’s Advice on COVID-19: You’re Stronger Than You Think

NORD Partners with ‘Sing Me a Story’ for July 18–20 Living Rare Forum

Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19

Critical Period of Vision Degeneration in CLN2 Patients 4 to 7 Years Old, Study Reveals

Anesthesia Generally Safe in Children and Adolescents with Batten Disease, Study Shows

Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds

Study Sheds Light on CLN3 Involvement in Juvenile Batten Disease

Elsevier Opens Books, Review Articles to Rare Disease Researchers Starting Feb. 29

My Rare Disease Day Challenge to Clinical-stage Companies

Advice on Starting Nonprofit Groups for Rare and Other Diseases Focus of NORD Webinar

Patients, Supporters Worldwide Recognizing Rare Disease Day 2020

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

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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

Subscribe to our mailing list

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

Axovant, Invitae Offering Free Genetic Testing, Counseling for Lysosomal Storage Diseases

FDA Favors Clinical Trial of Potential Juvenile Batten Treatment, PLX-200

NORD’s Advice on COVID-19: You’re Stronger Than You Think

NORD’s Advice on COVID-19: You’re Stronger Than You Think

NORD Partners with ‘Sing Me a Story’ for July 18–20 Living Rare Forum

Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19

For Many Rare Disease Families, Self-isolation Is Nothing New

Critical Period of Vision Degeneration in CLN2 Patients 4 to 7 Years Old, Study Reveals

Anesthesia Generally Safe in Children and Adolescents with Batten Disease, Study Shows

Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds

Video: BioNews’ Social Media Campaign Highlights #WhatMakesMeRare

Study Sheds Light on CLN3 Involvement in Juvenile Batten Disease

Elsevier Opens Books, Review Articles to Rare Disease Researchers Starting Feb. 29

My Rare Disease Day Challenge to Clinical-stage Companies

Advice on Starting Nonprofit Groups for Rare and Other Diseases Focus of NORD Webinar

Patients, Supporters Worldwide Recognizing Rare Disease Day 2020

Pin It on Pinterest