Batten Disease News

April 8, 2021April 8, 2021

Maypole Project Opens Counseling and Activities for Batten Families in UK

Batten Disease News

Families of a child with Batten disease in the U.K. are welcome take part in counseling and other wellness services being offered at no cost by the Maypole Project in a three-year collaboration with ... Read more

April 1, 2021April 1, 2021

Cost of Rare Diseases in US? Nearly $1 Trillion in 2019, EveryLife Foundation Finds

Batten Disease News

As anyone affected by a rare disease knows, treating the illness while trying to go about everyday life is an expensive undertaking.  But exactly how expensive — in terms of ... Read more

March 25, 2021March 25, 2021

Neurogene Workshop Seeks Families Affected by CLN5

Batten Disease News

Neurogene, which is developing treatments for Batten disease and other rare neurological disorders, is inviting families affected by CLN5, a form of late infantile Batten, to participate in a virtual caregiver ... Read more

March 11, 2021March 11, 2021

Actelion to Help Develop BBDF-101 for Juvenile Batten Disease

Batten Disease News

Beyond Batten Disease Foundation (BBDF) has struck a deal with Actelion Pharmaceuticals to help further the development of BBDF-101, an investigational treatment for juvenile Batten disease. Under the terms of ... Read more

March 8, 2021March 8, 2021

Pink, Green, Blue and Purple Lights Illuminated World for Rare Disease Day

Batten Disease News, COVID-19 News

Many diseases have their own awareness color — breast cancer is pink, muscular dystrophy is green, and AIDS is red, for example — but what’s the significance of pink, green, ... Read more

March 7, 2021March 18, 2021

Report: Atypical CLN2 Starts With Seizures, Language and Behavioral Problems

Batten Disease News

Atypical neuronal ceroid lipofuscinosis type 2 (CLN2) — a type of Batten disease that occurs later in childhood — presented with seizures, language difficulties, and behavioral problems as the first ... Read more

March 5, 2021March 5, 2021

Scientist Wins Grant to Study CLN6 Batten, Advance Early Trials

Batten Disease News

Sanford Research scientist Kevin Francis, PhD, has received an $83,718 research grant to study CLN6 Batten disease — also known as variant late infantile CLN6 disease — and advance Anavex’s ... Read more

February 26, 2021February 26, 2021

Rare Disease Day ‘Patient Hero’ Raises Awareness Through Fashion

Batten Disease News, COVID-19 News

Diagnosed with sickle cell disease as a 6-month-old, Tristan Lee has faced a lot of challenges over his 37 years of life. But from a young age, he also learned ... Read more

February 26, 2021February 26, 2021

Vision Loss in Juvenile Batten May Be Due to ‘Maintenance’ Failure

Batten Disease News

CLN3, the protein missing in people with juvenile Batten disease, is needed for a cellular process normally taking place in the retina that works to maintain the specialized cells needed ... Read more

February 22, 2021February 22, 2021

Rare Disease Day at NIH, Set for March 1, Growing Year by Year

Batten Disease News, COVID-19 News

Rare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place on March 1, will feature panel discussions, patient stories, research updates, TED-style talks, and a ... Read more

February 19, 2021February 19, 2021

Eurordis Survey: Healthcare Experience Worse for Rare Disease Patients

Batten Disease News

People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of an international survey conducted by Eurordis-Rare Diseases Europe. Indeed, rare ... Read more

February 19, 2021February 19, 2021

CLN3 Gene Therapy Showing Safety, Possible Efficacy in Trial, Amicus Reports

Batten Disease News

AT-GTX-502, Amicus Therapeutics’ investigational gene therapy for juvenile Batten disease, has been well-tolerated and is showing a potential to stabilize early signs of the disease in children and infants. These are ... Read more

February 12, 2021February 12, 2021

Gene Therapy Directly to Brain Helps Preserve Vision in CLN6 Mouse Model

Batten Disease News

A single injection into the brain of an investigational gene therapy now in clinical trials partly prevented retinal degeneration and vision loss in a mouse model of CLN6 Batten disease, a ... Read more

February 11, 2021February 11, 2021

Pandemic Won’t Stop Rare Disease Day on Feb. 28

Batten Disease News, COVID-19 News

Scores of virtual events are afoot around the world to mark Rare Disease Day 2021 on Feb. 28. The activities are focused on heightening awareness about rare diseases and the ... Read more

February 5, 2021February 5, 2021

Analysis of Proteins in Urine Identifies Potential Disease Biomarkers

Batten Disease News

A urine analysis, called a urine proteome, found higher expression of specific proteins in people with different subtypes of Batten disease, a study reports. These proteins may be useful as easily ... Read more