A photo of the Columbia Center in downtown Seattle lighting its crown in pink to support Rare Disease Day

I redownloaded Twitter just to grab this screenshot of a post by the Columbia Center social media team acknowledging Rare Disease Day. I promptly deleted the app again.

Lighting at the under-construction Mary Bridge Children’s Hospital in Tacoma displaying colors in support of Rare Disease Day

Lighting at the under-construction Mary Bridge Children’s Hospital in Tacoma displaying colors in support of Rare Disease Day

For Rare Disease Day (2/28), the amazing Jennifer Palermo reached out and got Seattle’s Columbia Center and the under-construction Mary Bridge Children’s Hospital in Tacoma to light up in Rare Disease Day colors!

Team Amelia Forever ❤️

#lightupforrare #rarediseaseday #raredisease #battendisease

Gene therapy could inform treatment for childhood dementia | The Roslin Institute | The Royal (Dick) School of Veterinary Studies Studies in mice and sheep offer hope for future treatment of a neurological disease affecting children.

Today, we are hosting families affected by Batten disease, a rare and severe childhood dementia.
Early-stage research has shown promising results for a gene therapy targeting this.
Mouse and sheep studies indicate potential benefits - an encouraging step forward.

#BattenDisease

edin.ac/4rrzctr

Childhood Dementia: Understanding the Silent Tragedy of Childhood 🧠 Childhood Dementia: when dementia strikes children🌟 A surprising nameWhen we hear the word dementi

www.myscrappydays.com/news.php?slu... — 🧠 Childhood Dementia: when dementia strikes children🌟 A surprising nameWhen we hear the word dementi… #Childhooddementia #Rarediseases #Battendisease #Sanfilipposyndrome #NiemannPickdisease #Geneticdisorders #Dementiainchildren #Neurodegenerativediseases

New publication working with lead Tristan McKay studying iPSC models to elucidate new functions for the CLN7 protein and its role in Batten Disease (Neuronal Ceroid Lipofuscinosis). Well done Aseel Sharaireh et al. #BattenDisease #neuroscience #iPSC @natureportfolio.nature.com
rdcu.be/eNIwd

New partnership targets gene therapy for vision loss in children... Tern and Andelyn are teaming up to advance the manufacturing of TTX-381, a gene therapy for vision loss in children with CLN2 Batten disease.

Exciting collaboration between biotech companies to advance gene therapy for Batten disease
battendiseasenews.com/news/partner...
#BattenDisease #GeneTherapy #BiotechInnovation #MedicalBreakthrough

Vacuolated lymphocytes: a diagnostic biomarker for CLN3-related Batten disease CLN3-related juvenile neuronal ceroid lipofuscinosis (JNCL) or Batten disease is a rare (1 in 25,000 to 3 in 100 000 livebirths) autosomal recessive neurometabolic storage disorder characterized by de...

CLN3-related juvenile neuronal ceroid lipofuscinosis (JNCL), or Batten disease, is a rare autosomal recessive neurometabolic storage disorder. This article details how vacuolated lymphocytes are a key diagnostic biomarker for this disease.

bit.ly/cjo_vacuolat...

#Ophthalmology #BattenDisease

Expressions of Interest OPEN for #BattenDisease Grant Round. The top priority research lines are so inspiring: best gene-targeted #therapy? which valid, reliable #biomarkers? Can disease progression be halted or reversed? Apply here: bdsrafoundation.org/wp-content/u...

Hope Restored in British Columbia Last Thursday, the British Columbia Ministry of Health announced that public funding for Brineura has been restored for 10-year-old Charleigh Pollock, who takes the medication to treat CLN2 Batten …

Today's blog post is about a reversed decision to provide medication to a child in Canada.

gracedowwrites.com/2025/07/21/h...
#Disability #Canada #BattenDisease

A Lifeline Is Gone On Friday, the British Columbia Ministry of Health announced that it will not reverse its decision to discontinue public funding for Brineura. 10-year-old Charleigh Pollock takes this medication to…

Today’s blog post is about a child being denied medication in Canada.

gracedowwrites.com/2025/07/13/a...
#Canada #Disability #BattenDisease

'Every day matters': Heartbroken B.C. mom urges government to restore drug funding for child with terminal brain disease Despite a personal plea from family, the province has upheld its decision to stop funding Brineura for nine-year-old Charleigh Pollock.

"Charleigh is the only person in B.C. diagnosed w/ #CLN2 #BattenDisease, a rare genetic #neurodegenerative disorder that progressively robs children of their ability to see, speak, walk, and eventually swallow." 👉 Example of #Canada #Healthcare failing those who need the most help.
#raredisease

As part of #BattenDisease Awareness Day we’re offering free access to expertly curated variant data for the CLN3 gene in #MastermindGIP 🧬 Our latest blog shares a story of a family searching for answers—and holding onto hope. See more & explore #CLN3: www.genomenon.com/blog/batten-...

You are our yellow 💛

A decade without this beautiful girl. I miss her so freaking much.

I love you, Mabel.

And I hate batten disease!!!!

#Yellow #BattenDisease #RareGenetics

It is honestly staggering how much medication our 8-year-old needs to sleep comfortably some nights (melatonin PLUS a bunch of others).

I hate #BattenDisease so very much.

Nothing in life is guaranteed. You can buy all the Little Tikes toys you want, but if your child was born with a horrible, neurodegenerative condition like #BattenDisease, they might just sit in the yard unused.

My life advice: Cherish life. Oh, and fund rare disease research.

Need to figure out how to get #BattenDisease research on his radar ASAP.

Amelia sits in bed with her BiPAP breathing apparatus on. She’s struggling to fall asleep due to intense gastrointestinal discomfort throughout the night, which is a common symptom for those with Batten Disease.

While #BattenDisease is often associated with the horrific damage it does to the brain, it also wreaks havoc on the GI tract, sometimes causing intense bouts of discomfort. Which is why this poor girl and her parents have been up til 6AM. Hopefully, we addressed the pain enough that she can sleep!

I haven’t slept in my own bed in two and a half years.

One of the many, many unexpected aspects of living in a #BattenDisease household!

Tom Wishart spoke at @edinunineuro.bsky.social #neuroday2025 this week on the use of large animal models for #biomarker discovery. Showcasing his work on #BattenDisease. #Research #NottinghamTrent

Redirecting...

Find out about some of the work being carried out in collaboration with our Neuro theme lead Prof Tom Wishart and colleagues on biomarkers of Batten Disease.

#BattenResearch #BattenDisease #RareDisease #Science #Research #Clinical #Grant

www.facebook.com/share/v/19gh...

Speech, Language and Non‐verbal Communication in CLN2 and CLN3 Batten Disease CLN2 and CLN3 diseases, the most common types of Batten disease (also known as neuronal ceroid lipofuscinosis), are childhood dementias associated with progressive loss of speech, language and feedin...

New research on CLN2 & CLN3 in Batten disease!
In collaboration with Murdoch Children's Research Institute, UoM, and others, we’ve uncovered insights into speech and cognitive decline.

onlinelibrary.wiley.com/doi/10.1002/...

#BattenDisease #CLN2 #CLN3 #NeurodegenerativeDiseases #Dementia

Donate to Charleigh's Journey, organized by Masika Woods **Update** We are all incredibly grateful for every dollar donated and are amazed at the generosity e… Masika Woods needs your support for Charleigh's Journey

www.gofundme.com/f/charleigh0...

Charleigh is a sweet little girl with #battendisease; a devestating, degenerative genetic condition that is typically fatal in childhood.
#albertahealth wants to cut off her treatments that sustain her quality of life.

Click below to read more.

On #RareDiseaseDay read about work by Tom Wishart of @ntuneuro.bsky.social & @roslininstitute.bsky.social on developing models and ultimately new treatments for rare childhood dementia condition, #BattenDisease. #RareDisease

vet.ed.ac.uk/roslin/news-...

Our experts have developed a robust method of studying Batten disease, a rare form of childhood dementia.

Their work is helping to understand the disease, and is enabling testing and development of therapies.

#RareDiseaseDay #RareDisease #BattenDisease

edin.ac/4hKv8iT

20 years ago today, I lost my 9 year old sister, Caitlin, to CLN2 Batten Disease. She was everything to me. If you'd like to know more about this cruel, very rare neurodegenerative disease, please consider visiting www.bdfa-uk.org.uk

Thank you 🧡 xx

#BattenDisease #CLN2BattenDisease

'Charleigh is going to die': Province pulls drug funding for nine-year-old with Batten Disease Nine-year-old with Batten Disease has treatment funding pulled by the province of B.C. despite more than five years of treatment.

So tragic for the family -
Almost $1million CAD/ year is a steep cost for medical treatment. Without it tho', " ‘Charleigh is going to die’ - Province pulls drug funding for nine-year-old with #BattenDisease"
cheknews.ca/charleigh-is...
#CharleighsJourney
#BCpoli #Cdnpoli #CdnHealthcare #FreeNorth

Identifying and treating CLN3 disease outside the central nervous system CLN3 disease causes profound neurological deficits in affected children, but less well recognized are a variety of peripheral neuromuscular and gastrointestinal problems. We hypothesized that in addit...

Pleased to share the latest pre-print from the PSDL highlighting enteric and neuromuscular disease & myopathy in CLN3 mice treated by gene therapy. From talented PSDL postdoc Ewa Ziółkowska. Work done with Bob Heuckeroth and Alison Snyder-Warwick #battendisease
www.biorxiv.org/content/10.1...

Looking forward to the 2025 WORLDSymposium on Lysosomal Diseases starting tomorrow. Ewa Ziółkowska (PSDL postdoc) has won a very well-deserved Young Investigator Award and will present her work on dysphagia (swallowing problems) #battendisease

A year ago, we almost lost our Amelia after a bout of the flu put her on life support for 3 weeks. #BattenDisease families know time is short, but we weren’t ready to say goodbye. Thankfully, her lungs improved, though they’re clearly still diminished.

youtube.com/shorts/5lECk...

Amelia, the CLN1 Warrior! - Rare Disease Day 2025 Our daughter, Amelia, was diagnosed with CLN1 Batten Disease (neuronal ceroid lipofuscinosis) at 2 years old. Batten Disease is a fatal, neurodegenerative disease with no… Continue reading Amelia, the...

The kind folks at Rare Disease Day have shared Amelia’s #BattenDisease story, and you can read it at the link below!

Rare Disease Day is Feb. 28th and the more awareness we can spread, the better!

www.rarediseaseday.org/heroes/ameli...