Updates

June 14, 2019June 14, 2019
Fycompa May Help Control Involuntary Muscle Jerks in Patients With CLN2 Disease, According to Case Report

Fycompa (perampanel) may be considered as an add-on therapy to control myoclonus (quick, involuntary muscle jerks) in patients with late infantile Batten disease, according ... Read more
June 7, 2019June 7, 2019
Blood Spot Test May Aid Early Diagnosis of Infantile Batten Disease, Study Says

A new dried blood spot test may be a reliable and rapid method for obtaining an initial diagnosis of late infantile Batten disease, recent ... Read more
May 31, 2019May 31, 2019
Nationwide Children’s Hospital Names Lobby in Honor of Gray Foundation

Nationwide Children’s Hospital, the site of a first-of-its-kind clinical trial in Batten disease, has dedicated its lobby in honor of the Charlotte and Gwenyth ... Read more
May 29, 2019May 28, 2019
My Road to Big Victories for CLN1 Disease

With 350 million patients, rare diseases represent the largest disease community on the planet. In fact, if all rare disease patients lived in the ... Read more
May 27, 2019May 24, 2019
Agency Unveils RaDaR to Help Patient Groups Develop Rare Disease Registries

RaDaR, the catchy new name for the U.S. government-run Rare Diseases Registry Program, aims to help patient advocacy groups with limited resources build their ... Read more

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News

FDA Clears ABO-202 Gene Therapy Candidate for Clinical Trials in Infantile Batten Disease
May 24, 2019May 24, 2019
The U.S. Food and Drug Administration (FDA) accepted Abeona Therapeutics’ investigational new drug application ...
News

Rare Diseases Constitute a ‘Public Health Issue,’ NCATS Director Warns
May 15, 2019May 15, 2019
With 250 rare diseases newly identified every year, scientists can barely keep up — ...
News

CLN3 Deficiency Leads to Abnormalities in Cellular Recycling Process, Study Shows
May 10, 2019May 10, 2019
The CLN3 gene may play a more crucial role than previously thought in the ...
News

Loss of TPP1 Linked to Brain Inflammation and Nerve Cells’ Dysfunction, Mouse Study Says
May 3, 2019May 3, 2019
Loss of the Tpp1 gene is associated with brain inflammation and nerve cell dysfunction, possibly due ...
News

Patients Showing Signs of LINCL Should be Tested for CLN5, CLN8 Mutations, Study Says
April 26, 2019April 26, 2019
Patients experiencing typical symptoms of late-infantile neuronal ceroid lipofuscinoses (LINCL) should be tested for the presence ...
News

Nonprofit Works to Raise Rare Disease Awareness in India, Connect with U.S. Groups
April 25, 2019April 25, 2019
With an estimated 1.37 billion inhabitants, India will likely surpass China in five years ...

Scientists Discover New Mutation in CLN3 Gene Associated with Juvenile Batten Disease
April 12, 2019April 12, 2019
Scientists have discovered a new mutation in the CLN3 gene associated with Juvenile Batten ...
Brineura Over 3 Years Seen to Delay Loss of Motor-Language Skills in CLN2 Children, Trial Data Shows
April 5, 2019April 5, 2019
Three years of treatment with Brineura (cerliponase alfa) was seen to effectively and durably delay the progression ...
New Therapies Being Developed for Batten Disease at ‘Unprecedented’ Pace, Review Suggests
March 29, 2019March 29, 2019
Alpha-Synuclein Protein May Contribute Toward Batten Disease Progression
March 22, 2019March 22, 2019
Brexit Could Have Real Effects for UK Rare Disease Patients, Experts Warn
March 15, 2019March 14, 2019
Moral Dilemmas Complicate Treatment of Rare Diseases, Says Israeli Bioethicist
March 11, 2019March 11, 2019

Amicus Research and Gene Therapy Center To Focus on Batten, Other Diseases
March 8, 2019March 8, 2019
To advance its commitment to research aimed at improving the lives of those with ...
NIH Rare Disease Day Highlights Joint Networks Advancing Array of Research
March 5, 2019March 5, 2019
WODC 2019 Organizers Expect 1,200 to Attend Rare Disease Conference in April
February 25, 2019February 25, 2019
Vienna to Host RARE2019 Meeting on Rare Diseases
February 22, 2019February 20, 2019

Patients, Family and Friends to Take Part in Variety of Rare Disease Day Events Worldwide
February 11, 2019February 11, 2019
Scores of events are afoot worldwide to mark Feb. 28, Rare Disease Day 2019. The activities ...
One-time Gene Therapy RGX-181 Wins FDA Rare Pediatric Disease Designation
February 8, 2019February 8, 2019
ICD-10 Codes, ‘Really Important’ to Rare Disease Patients, Soon Up for Fresh Consideration
February 6, 2019February 6, 2019

News

Mouse Model for CLN6 Disease Shows Sex-specific Differences in Behavior, Lifespan, Study Finds
February 1, 2019February 1, 2019
A mouse model representing CLN6 disease, a subtype of Batten disease, shows that females ...
News

FDA Revising ‘Draft Guidance’ on Developing Treatments for Rare Diseases
January 30, 2019January 30, 2019
The U.S. Food and Drug Administration(FDA) is updating its 2015 draft guidelines for drug ...
News

Mutated CLN3 Protein May Indirectly Promote Abnormal Signaling Cascades Involved in Juvenile Batten Disease, Study Finds
January 25, 2019January 25, 2019
Mutated forms of CLN3 protein may be involved in the abnormal activation of signals ...
News

CLN3 Disease Should be Considered When Diagnosing AVM, Researchers Say
January 18, 2019January 18, 2019
A team of Italian researchers suggests that CLN3 disease, also known as juvenile Batten ...
News

Both Rare and Classic Juvenile Batten Types Show Changes in Skeletal Muscle Cells, Study Finds
January 11, 2019January 11, 2019
The activation of a recycling pathway, a process called autophagy, which leads to large ...
News

Amicus Therapeutics Recruiting Children with CLN3 Batten Disease for Gene Therapy Trial
January 4, 2019January 4, 2019
Amicus Therapeutics is recruiting children for its new Phase 1/2 clinical trial that will ...

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

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