NORD Partners with ‘Sing Me a Story’ for July 18–20 Living Rare Forum
The Living Rare, Living Stronger Patient and Family Forum, originally set for May 14–16 in Cleveland, Ohio, has been postponed until July 18–20 because ... Read more
Updates
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Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19
Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia Monaco, PhD, of Italy remains confident that the Paris-based ... Read more -
For Many Rare Disease Families, Self-isolation Is Nothing New
We watched from the dock as sunlight danced on the lake’s surface — a balletic performance for a small but captive audience of one ... Read more -
Critical Period of Vision Degeneration in CLN2 Patients 4 to 7 Years Old, Study Reveals
Vision degeneration in children with CLN2 disease, also known as late infantile Batten disease, occurs similarly in both eyes and seems to accelerate from ages ... Read more -
Anesthesia Generally Safe in Children and Adolescents with Batten Disease, Study Shows
Anesthesia seems generally safe in children and adolescents with Batten disease, according to a study evaluating complications immediately before, during, and after procedures that ... Read more
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Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds
The number of treatments for children with rare diseases has grown over the past decade, ... -
Video: BioNews’ Social Media Campaign Highlights #WhatMakesMeRare
In recognition of Rare Disease Day Feb. 29, BioNews Services launched a social media ... -
Study Sheds Light on CLN3 Involvement in Juvenile Batten Disease
The CLN3 protein — the defective form of which underlies juvenile Batten disease — ... -
Elsevier Opens Books, Review Articles to Rare Disease Researchers Starting Feb. 29
Beginning on Feb. 29, Rare Disease Day, chapters from notable scientific books and clinical review ... -
My Rare Disease Day Challenge to Clinical-stage Companies
The world will soon celebrate Rare Disease Day. The 2020 edition falls on Feb. ... -
Advice on Starting Nonprofit Groups for Rare and Other Diseases Focus of NORD Webinar
Starting a 501(c)(3) tax-exempt nonprofit isn’t easy, but the National Organization for Rare Disorders ...
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