Updates

October 11, 2019October 11, 2019
Customized Oligonucleotides Treat Seizures in Child with CLN7 Disease in Record Time

Neurologists and researchers have tailor-made an antisense oligonucleotide therapy (ASO) for a 6-year-old girl who has CLN7 Batten disease, and it cut to half her number ... Read more
October 4, 2019October 4, 2019
UFC Cheers Young Fan with Batten While Helping to Raise Disease Awareness

The Ultimate Fighting Championship (UFC) lifted the spirits of a boy named Sebastian recently while helping to make everyone more aware of Batten disease, the ... Read more
October 2, 2019September 30, 2019
9 Things We Should Know About the Race to New Treatments

I favor running analogies — even the name of this column pays tribute to the sport. But I’m not the first patient advocate to ... Read more
September 27, 2019September 26, 2019
NORD 2019 Rare Disease Summit Set for Oct. 21-22 in Washington, DC

Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a better time, says Marshall Summar, ... Read more
September 20, 2019September 20, 2019
UK Batten Families Welcome Brineura Access for CLN2 Patients Through NHS

When Ollie Carroll was 5 years old, the British boy made history by becoming one of the world’s youngest Batten disease patients to begin ... Read more

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News

Taylor’s Tale Co-founder Races to ‘Change the World’ for Batten Disease Community
September 6, 2019September 9, 2019
Laura King Edwards ran a half-marathon as a fifth anniversary nod to her sister’s first ...
News

Extracellular Vesicles May Enhance Delivery of TPP1 to the Brain for Replacement Therapy in CLN2, Early Study Suggests
August 30, 2019August 30, 2019
Extracellular vesicles derived from a type of immune cell called macrophage may hold potential ...
News

‘Disorder’ Film Festival Offers Look at Siblings Touched by Rare Diseases
August 30, 2019August 30, 2019
Rare diseases deeply affect not only the children who experience them, but also their ...
News

At NY Genome Center, Legal Expert Presents Ethical Dilemmas in Gene Editing
August 29, 2019August 28, 2019
Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is ...
Columns, The Long Sprint - a Column by Laura King Edwards

Can Patients and Families Really Drive Therapy Development?
August 28, 2019August 28, 2019
The therapy development process is a marathon, not a sprint. But unlike a marathon, ...
News

Amicus CEO John Crowley Outlines Progress in Batten, Pompe, Fabry Therapies
August 23, 2019August 21, 2019
Pharmaceutical executives rarely make for a sympathetic Hollywood medical drama. But John Crowley did, ...

Nebraska’s Neena Nizar Seeks Cure for Jansen’s, One of World’s Rarest Diseases
August 20, 2019
Imagine living your whole life with a painful disease so rare that only 25 ...
Neurogene Opens Natural History Study of CLN7 and CLN5 Disease at UT Southwestern
August 9, 2019August 9, 2019
Neurogene is sponsoring a natural history study to better understand the course of CLN5 ...
Oklahoma Ranks Lowest on Programs Key to Rare Diseases on NORD’s 2019 State Report Card
August 2, 2019August 1, 2019
Rare Disease Groups Seek Public Support to Renew Newborn Screening Act in Senate
August 1, 2019
Amicus, Paragon to Collaborate on Gene Therapies for Batten, Other Diseases
July 26, 2019July 26, 2019
European Initiative Targets Diagnosis, Treatment of Rare Diseases
July 19, 2019July 18, 2019

Striking a Balanced View of Clinical Trials
July 17, 2019July 15, 2019
This month marks 13 years since my sister’s diagnosis of CLN1 disease, or Batten ...
Brain-Directed Gene Therapy May Prevent Neurodegeneration in CLN6 Disease, Study Says
July 12, 2019July 12, 2019
World’s First Alport Stamp Is Macedonian Mom’s Latest Win for Rare Disease Patients
July 4, 2019July 4, 2019
Motor Impairments Are Early Sign of Juvenile Batten Disease, Study Finds
June 28, 2019June 28, 2019

3.1 Ways to Fight Your Rare Disease
June 26, 2019June 25, 2019
My life changed forever 13 years ago. In the space of a few months, ...
NORD Honors Industry, Patient Advocates at Rare Impact Awards Gala
June 26, 2019June 25, 2019
FDA Grants Fast Track Designation to ABO-202 Gene Therapy for Infantile Batten Disease
June 21, 2019June 21, 2019

News

Eurordis Unveils Integrated-care Initiative for Rare Disease Patients
June 21, 2019June 20, 2019
Europe’s umbrella organization for 800 rare disease associations has developed a sweeping initiative to ...
News

‘Rare Barometer’ Program Helps Eurordis Shape EU Rare Disease Policy
June 17, 2019June 17, 2019
People with rare diseases know that the right government policies can make a big ...
News

Fycompa May Help Control Involuntary Muscle Jerks in Patients With CLN2 Disease, According to Case Report
June 14, 2019June 14, 2019
Fycompa (perampanel) may be considered as an add-on therapy to control myoclonus (quick, involuntary ...
News

Blood Spot Test May Aid Early Diagnosis of Infantile Batten Disease, Study Says
June 7, 2019June 7, 2019
A new dried blood spot test may be a reliable and rapid method for ...
News

Nationwide Children’s Hospital Names Lobby in Honor of Gray Foundation
May 31, 2019May 31, 2019
Nationwide Children’s Hospital, the site of a first-of-its-kind clinical trial in Batten disease, has ...
Columns, The Long Sprint - a Column by Laura King Edwards

My Road to Big Victories for CLN1 Disease
May 29, 2019May 28, 2019
With 350 million patients, rare diseases represent the largest disease community on the planet. ...

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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