Updates

August 23, 2019August 21, 2019
Amicus CEO John Crowley Outlines Progress in Batten, Pompe, Fabry Therapies

Pharmaceutical executives rarely make for a sympathetic Hollywood medical drama. But John Crowley did, and in the nearly 10 years since the release of ... Read more
August 20, 2019
Nebraska’s Neena Nizar Seeks Cure for Jansen’s, One of World’s Rarest Diseases

Imagine living your whole life with a painful disease so rare that only 25 others worldwide have what you have. And that you’re one ... Read more
August 9, 2019August 9, 2019
Neurogene Opens Natural History Study of CLN7 and CLN5 Disease at UT Southwestern

Neurogene is sponsoring a natural history study to better understand the course of CLN5 and CLN7 disease, two forms of late-infantile Batten disease, and ... Read more
August 2, 2019August 1, 2019
Oklahoma Ranks Lowest on Programs Key to Rare Diseases on NORD’s 2019 State Report Card

Oklahoma suffers more tornadoes than any other state, has the highest per-capita rate of women in U.S. prisons, ranks second in the number of ... Read more
August 1, 2019
Rare Disease Groups Seek Public Support to Renew Newborn Screening Act in Senate

Screening newborns for genetic diseases with treatments that can prevent crippling or deadly progression, especially for rare disorders, has a ways to go in ... Read more

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News

Amicus, Paragon to Collaborate on Gene Therapies for Batten, Other Diseases
July 26, 2019July 26, 2019
Amicus Therapeutics entered a collaboration with Paragon Gene Therapy, a unit of Catalent Biologics, ...
News

European Initiative Targets Diagnosis, Treatment of Rare Diseases
July 19, 2019July 18, 2019
A new international consortium based in Paris, and funded largely by the 28-member European ...
Columns, The Long Sprint - a Column by Laura King Edwards

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 ...
News

Brain-Directed Gene Therapy May Prevent Neurodegeneration in CLN6 Disease, Study Says
July 12, 2019July 12, 2019
Brain-directed gene therapy is a feasible therapeutic strategy to prevent neurodegeneration associated with CLN6 ...
News

World’s First Alport Stamp Is Macedonian Mom’s Latest Win for Rare Disease Patients
July 4, 2019July 4, 2019
It wasn’t until Gordana Loleska’s son David was 14 years old that doctors in ...
News

Motor Impairments Are Early Sign of Juvenile Batten Disease, Study Finds
June 28, 2019June 28, 2019
In most patients with juvenile Batten disease, motor impairments are already apparent at the ...

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
A violinist with vasculitis, two Texas politicians and a pharmaceutical company whose marijuana-derived therapy ...
FDA Grants Fast Track Designation to ABO-202 Gene Therapy for Infantile Batten Disease
June 21, 2019June 21, 2019
Eurordis Unveils Integrated-care Initiative for Rare Disease Patients
June 21, 2019June 20, 2019
‘Rare Barometer’ Program Helps Eurordis Shape EU Rare Disease Policy
June 17, 2019June 17, 2019
Fycompa May Help Control Involuntary Muscle Jerks in Patients With CLN2 Disease, According to Case Report
June 14, 2019June 14, 2019

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 ...
Nationwide Children’s Hospital Names Lobby in Honor of Gray Foundation
May 31, 2019May 31, 2019
My Road to Big Victories for CLN1 Disease
May 29, 2019May 28, 2019
Agency Unveils RaDaR to Help Patient Groups Develop Rare Disease Registries
May 27, 2019May 24, 2019

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 ...
Rare Diseases Constitute a ‘Public Health Issue,’ NCATS Director Warns
May 15, 2019May 15, 2019
CLN3 Deficiency Leads to Abnormalities in Cellular Recycling Process, Study Shows
May 10, 2019May 10, 2019

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 ...
News

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 ...
News

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 ...
News

New Therapies Being Developed for Batten Disease at ‘Unprecedented’ Pace, Review Suggests
March 29, 2019March 29, 2019
Substantial progress has been made by different academic and industry teams toward the development of ...

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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