August 22, 2017August 22, 2017
Abeona Will Give Update on Its Gene Therapy Programs in R&D Day Set for October

Abeona Therapeutics will host an inaugural Research and Development (R&D) Day in New York City in October, briefing investors ... Read more
August 16, 2017
Batten Disease Clinical Trials Enrolling Patients to Evaluate Gene Therapies

Gene therapies for children with Batten disease are being evaluated in two clinical trials — one in New York and ... Read more
August 8, 2017
Polaryx’s Treatment Candidate for LINCL Granted FDA Orphan Drug Status

The U.S. Food and Drug Administration (FDA) granted orphan drug status to Polaryx Therapeutics’ PLX-200 in response to a ... Read more
August 3, 2017
Charlotte and Gwenyth Gray Foundation Funds Clinical Trial for Rare Batten Disease Variant

The Charlotte and Gwenyth Gray Foundation to Cure Batten Disease was launched in 2015 by Hollywood producer Gordon Gray ... Read more
August 1, 2017
National Organization for Rare Disorders Gives Batten Disease Community a Voice in Washington

Even by the standards of rare illnesses, Batten disease is extremely uncommon, affecting only two to four of every ... Read more
July 27, 2017July 27, 2017
Current Batten Therapeutics Targeting Brain Not Enough to Stop Disease in Other Organs, Canine Study Suggests

Current therapeutics, including gene therapy, that are based on delivery of TTP1 protein directly into the central nervous system ... Read more
July 25, 2017
CLN5 Protein Loss in Batten Disease Led to Fewer Functional Neurons in Mouse Study

Researchers studying the loss of the CLN5 protein, which causes Batten disease, found that it leads to the reduced ... Read more
July 20, 2017July 20, 2017
North Carolina’s Indo Jax Surf Charities Helps Kids With Batten Disease, Other Special Needs

A small North Carolina nonprofit organization, Indo Jax Surf Charities, has recently gained attention for its set of free ... Read more
July 13, 2017
Infantile Batten Disease May Begin in Spinal Cord Rather Than Brain, Study Suggests

New research shows that patients with infantile Batten disease exhibit significant early disease in the spinal cord and that ... Read more
July 11, 2017
Young Batten’s Patient Inspires Lawmakers to Create Rare Disease Advisory Council

Pennsylvania Gov. Tom Wolf recently signed House Bill 239, establishing the Rare Disease Advisory Council. The bill, was introduced May ... Read more
July 6, 2017
EU and US Regulators Offer Recommendations on Developing Therapies for Children’s Diseases

European and U.S. regulators have released a white paper on ways to develop innovative therapies for Gaucher disease that could also ... Read more
June 30, 2017June 30, 2017
FDA Marks Potential Gene Therapy for Juvenile Batten Disease as Orphan Drug to Speed Development

Abeona Therapeutics’ ABO-201 program was designed at orphan drug by the U.S. Food and Drug Administration (FDA) as a ... Read more

Abeona Will Give Update on Its Gene Therapy Programs in R&D Day Set for October

Abeona Therapeutics will host an inaugural Research and Development (R&D) Day in New York City in October, briefing investors on its progress in developing gene therapies for rare diseases, ... Read more

August 16, 2017

Batten Disease Clinical Trials Enrolling Patients to Evaluate Gene Therapies

Gene therapies for children with Batten disease are being evaluated in two clinical trials — one in New York and the other in Ohio. The trials are testing ways to ... Read more

August 8, 2017

Polaryx’s Treatment Candidate for LINCL Granted FDA Orphan Drug Status

The U.S. Food and Drug Administration (FDA) granted orphan drug status to Polaryx Therapeutics’ PLX-200 in response to a lack of treatments available for late infantile neuronal ceroid lipofuscinosis ... Read more

August 3, 2017

Charlotte and Gwenyth Gray Foundation Funds Clinical Trial for Rare Batten Disease Variant

The Charlotte and Gwenyth Gray Foundation to Cure Batten Disease was launched in 2015 by Hollywood producer Gordon Gray and his wife Kristen to advocate and raise funds for ... Read more

August 1, 2017

National Organization for Rare Disorders Gives Batten Disease Community a Voice in Washington

Even by the standards of rare illnesses, Batten disease is extremely uncommon, affecting only two to four of every 100,000 births in the United States. That translates into 20 ... Read more

July 27, 2017July 27, 2017

Current Batten Therapeutics Targeting Brain Not Enough to Stop Disease in Other Organs, Canine Study Suggests

Current therapeutics, including gene therapy, that are based on delivery of TTP1 protein directly into the central nervous system of patients with Batten disease may not be enough to ... Read more

July 25, 2017

CLN5 Protein Loss in Batten Disease Led to Fewer Functional Neurons in Mouse Study

Researchers studying the loss of the CLN5 protein, which causes Batten disease, found that it leads to the reduced production of new fully functional neurons. It also increased neuronal ... Read more

July 20, 2017July 20, 2017

North Carolina’s Indo Jax Surf Charities Helps Kids With Batten Disease, Other Special Needs

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 ... Read more

July 13, 2017

Infantile Batten Disease May Begin in Spinal Cord Rather Than Brain, Study Suggests

New research shows that patients with infantile Batten disease exhibit significant early disease in the spinal cord and that combined treatment of the spinal cord and the brain has ... Read more

July 11, 2017

Young Batten’s Patient Inspires Lawmakers to Create Rare Disease Advisory Council

Pennsylvania Gov. Tom Wolf recently signed House Bill 239, establishing the Rare Disease Advisory Council. The bill, was introduced May 24 by state Rep. Marcy Toepel, R-Montgomery County, who said the ... Read more

July 6, 2017

EU and US Regulators Offer Recommendations on Developing Therapies for Children’s Diseases

European and U.S. regulators have released a white paper on ways to develop innovative therapies for Gaucher disease that could also apply to Batten disease and other rare children’s disorders. The ... Read more

June 30, 2017June 30, 2017

FDA Marks Potential Gene Therapy for Juvenile Batten Disease as Orphan Drug to Speed Development

Abeona Therapeutics’ ABO-201 program was designed at orphan drug by the U.S. Food and Drug Administration (FDA) as a potential gene therapy treatment for juvenile Batten disease. A clinical ... Read more

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