Batten Disease News

April 24, 2020April 24, 2020

Brineura Shows Promise Delaying Disease Progression in Children with CLN2, Review Suggests

Batten Disease News

Brineura (cerliponase alfa), the first and only approved treatment for CLN2 disease, or late infantile Batten disease, is effective in delaying motor and language decline in children with the disease, ... Read more

April 22, 2020April 22, 2020

Rare Disease Therapy Development: Finding a Way Forward

Batten Disease News

Gaining access to lifesaving or even life-altering treatments has never been easy for many rare disease patients — including those with Batten disease. But the landscape is changing. What felt ... Read more

April 17, 2020April 17, 2020

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

Batten Disease News

Axovant Gene Therapies is collaborating with Invitae to offer free genetic testing in the United States and Canada to children suspected of having a lysosomal storage disease. The Axovant sponsorship ... Read more

April 10, 2020April 10, 2020

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

Batten Disease News

Polaryx Therapeutics has received approval from the U.S. Food and Drug Administration (FDA) to open a clinical trial into PLX-200 as a potential treatment for juvenile Batten disease. Juvenile Batten, also ... Read more

April 2, 2020May 2, 2020

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

Batten Disease News

First, the bad news: If you’re one of the 30 million or so Americans with a rare disease, you probably have lower immunity to the novel coronavirus than most people. ... Read more

March 27, 2020May 15, 2020

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

Batten Disease News, COVID-19 News

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 of the coronavirus disease COVID-19 pandemic. The ... Read more

March 25, 2020May 8, 2020

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

Batten Disease News, COVID-19 Columns

We watched from the dock as sunlight danced on the lake’s surface — a balletic performance for a small but captive audience of one in the quiet cove. Though still ... Read more

March 19, 2020March 21, 2020

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

Batten Disease News

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 4 to 7, according to a ... Read more

March 12, 2020March 21, 2020

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

Batten Disease News

Anesthesia seems generally safe in children and adolescents with Batten disease, according to a study evaluating complications immediately before, during, and after procedures that require anesthesia. These complications, however, were ... Read more

March 9, 2020March 21, 2020

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

Batten Disease News

The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking ... Read more

March 5, 2020March 21, 2020

Video: BioNews’ Social Media Campaign Highlights #WhatMakesMeRare

Batten Disease News

In recognition of Rare Disease Day Feb. 29, BioNews Services launched a social media campaign last month asking patients to describe what makes them rare. Running Feb. 7–29, the #WhatMakesMeRare ... Read more

March 5, 2020March 21, 2020

Study Sheds Light on CLN3 Involvement in Juvenile Batten Disease

Batten Disease News

The CLN3 protein — the defective form of which underlies juvenile Batten disease — regulates several degradation and recycling processes within cells, mainly associated with a protein called Rab7A, a ... Read more

March 2, 2020March 21, 2020

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

Batten Disease News

Beginning on Feb. 29, Rare Disease Day, chapters from notable scientific books and clinical review articles covering rare disorders will be available free-of-charge from Elsevier. The offer runs through April 30, and ... Read more

February 28, 2020March 21, 2020

My Rare Disease Day Challenge to Clinical-stage Companies

Batten Disease News

The world will soon celebrate Rare Disease Day. The 2020 edition falls on Feb. 29 — a rare day, indeed. EURORDIS founded Rare Disease Day in 2008 to raise awareness ... Read more

February 27, 2020March 21, 2020

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

Batten Disease News

Starting a 501(c)(3) tax-exempt nonprofit isn’t easy, but the National Organization for Rare Disorders gave a few tips for those  looking to begin the complex process in its Feb. 20 ... Read more