Batten Disease News

June 17, 2021June 17, 2021

Get Ready for NORD’s Patient and Family Forum, Set for June 26-27

Those who wish to gain practical tools for living optimally with rare diseases are encouraged to attend the annual Living Rare Living Stronger Patient and Family Forum, hosted by the ... Read more

June 17, 2021June 17, 2021

Brineura Delays CLN2 Onset, Progression in Case Study of 2 Siblings

Batten Disease News

Two years of treatment with Brineura (cerliponase alfa) safely and effectively delayed the onset of late-infantile Batten disease in a 23-month-old boy who had not exhibited symptoms when he started ... Read more

June 10, 2021June 10, 2021

Rare 2030 Action Campaign Pushing for European Plan for Rare Diseases

Batten Disease News

Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in ... Read more

June 4, 2021June 4, 2021

Advocates Can Try for Grants Ahead of Rare Disease Week on Capitol Hill

Batten Disease News

Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will award grants to top advocates of rare disease organizations who participate in ... Read more

June 3, 2021June 3, 2021

International Batten Disease Awareness Day is June 9

Batten Disease News

From participating in a virtual 5K to sharing family stories and videos, supporters are gearing up for International Batten Disease Awareness Day on June 9. The goal is to heighten ... Read more

May 27, 2021May 27, 2021

New Guidelines Issued for CLN2 Diagnosis, Management

Batten Disease News

An international team of experts has provided the latest evidence-based recommendations for diagnosis, assessment, management, and treatment of neuronal ceroid lipofuscinosis type 2 (CLN2), a study reported. The study, “Guidelines on ... Read more

May 20, 2021May 20, 2021

Cutting-edge Research Awarded BDSRA Australia’s 2020 Grants

Batten Disease News

The Batten Disease Support and Research Association (BDSRA) Australia has announced the winners of the 2020 Batten disease research grant program in Australia and New Zealand. The purpose of the program ... Read more

May 13, 2021May 13, 2021

Updates Delayed on Regenxbio’s CLN2 Gene Therapy Programs

Batten Disease News

Regenxbio plans to provide updates on its RGX-181 and RGX-381 gene therapy candidates for late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, one of the most common forms of Batten disease, ... Read more

May 6, 2021May 6, 2021

Brineura May Stop Progression in Typical, Atypical CLN2

Batten Disease News

Brineura (cerliponase alfa) stabilized the progression of neuronal ceroid lipofuscinosis type 2 (CLN2) — a type of Batten disease — in a small group of people in Columbia, including those ... Read more

April 30, 2021April 30, 2021

Caregivers of Children With CLN5 Sought for Neurogene Study Interviews

Batten Disease News

Neurogene, a biopharmaceutical company developing therapies for Batten disease, is trying to learn more about the progression of the subtype CLN5 disease from the caregiver perspective. To that end, Neurogene ... Read more

April 22, 2021April 22, 2021

Quick Approval Given COVID Vaccines Raise Concerns, Rare Disease Patients Say

Batten Disease News, COVID-19 News

More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less than willing to be vaccinated for COVID-19 if a vaccine ... Read more

April 15, 2021April 15, 2021

EveryLife Scholarship Fund Open Until May 7 for Adults With Rare Diseases in US

Batten Disease News, COVID-19 News

The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a rare disease pursue personal goals through training and education. For a ... Read more

April 8, 2021April 8, 2021

Maypole Project Opens Counseling and Activities for Batten Families in UK

Batten Disease News

Families of a child with Batten disease in the U.K. are welcome take part in counseling and other wellness services being offered at no cost by the Maypole Project in a three-year collaboration with ... Read more

April 1, 2021April 1, 2021

Cost of Rare Diseases in US? Nearly $1 Trillion in 2019, EveryLife Foundation Finds

Batten Disease News

As anyone affected by a rare disease knows, treating the illness while trying to go about everyday life is an expensive undertaking. But exactly how expensive — in terms of ... Read more

March 25, 2021March 25, 2021

Neurogene Workshop Seeks Families Affected by CLN5

Batten Disease News

Neurogene, which is developing treatments for Batten disease and other rare neurological disorders, is inviting families affected by CLN5, a form of late infantile Batten, to participate in a virtual caregiver ... Read more