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
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
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.
edin.ac
November 28, 2025 at 2:43 PM
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
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
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
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
November 10, 2025 at 6:58 PM
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
A question on #CerliponaseAlfa #BattenDisease tabled by Warinder Juss on 21-10-2025 has been answered by Dr Zubir Ahmed. https://questions-statements.parliament.uk/written-questions/detail/2025-10-21/84114
November 6, 2025 at 11:20 AM
A question on #CerliponaseAlfa #BattenDisease tabled by Warinder Juss on 21-10-2025 has been answered by Dr Zubir Ahmed. https://questions-statements.parliament.uk/written-questions/detail/2025-10-21/84114
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
rdcu.be/eNIwd
November 2, 2025 at 11:34 PM
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
rdcu.be/eNIwd
Exciting collaboration between biotech companies to advance gene therapy for Batten disease
battendiseasenews.com/news/partner...
#BattenDisease #GeneTherapy #BiotechInnovation #MedicalBreakthrough
battendiseasenews.com/news/partner...
#BattenDisease #GeneTherapy #BiotechInnovation #MedicalBreakthrough
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.
battendiseasenews.com
September 8, 2025 at 8:17 PM
Exciting collaboration between biotech companies to advance gene therapy for Batten disease
battendiseasenews.com/news/partner...
#BattenDisease #GeneTherapy #BiotechInnovation #MedicalBreakthrough
battendiseasenews.com/news/partner...
#BattenDisease #GeneTherapy #BiotechInnovation #MedicalBreakthrough
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
bit.ly/cjo_vacuolat...
#Ophthalmology #BattenDisease
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...
bit.ly
September 8, 2025 at 2:37 PM
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
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...
bdsrafoundation.org
August 21, 2025 at 9:11 AM
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...
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
gracedowwrites.com/2025/07/21/h...
#Disability #Canada #BattenDisease
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 …
gracedowwrites.com
July 21, 2025 at 5:54 AM
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
gracedowwrites.com/2025/07/21/h...
#Disability #Canada #BattenDisease
Today’s blog post is about a child being denied medication in Canada.
gracedowwrites.com/2025/07/13/a...
#Canada #Disability #BattenDisease
gracedowwrites.com/2025/07/13/a...
#Canada #Disability #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…
gracedowwrites.com
July 13, 2025 at 10:35 AM
Today’s blog post is about a child being denied medication in Canada.
gracedowwrites.com/2025/07/13/a...
#Canada #Disability #BattenDisease
gracedowwrites.com/2025/07/13/a...
#Canada #Disability #BattenDisease
"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
#raredisease
'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.
vancouversun.com
July 6, 2025 at 5:57 PM
"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
#raredisease
A question on #BattenDisease #CerliponaseAlfa tabled by Lord Botham on 23-06-2025 has been answered by Baroness Merron. https://questions-statements.parliament.uk/written-questions/detail/2025-06-23/HL8689
July 1, 2025 at 3:20 PM
A question on #BattenDisease #CerliponaseAlfa tabled by Lord Botham on 23-06-2025 has been answered by Baroness Merron. https://questions-statements.parliament.uk/written-questions/detail/2025-06-23/HL8689
A question on #BattenDisease #CerliponaseAlfa tabled by Lord Botham on 23-06-2025 has been answered by Baroness Merron. https://questions-statements.parliament.uk/written-questions/detail/2025-06-23/HL8690
July 1, 2025 at 3:20 PM
A question on #BattenDisease #CerliponaseAlfa tabled by Lord Botham on 23-06-2025 has been answered by Baroness Merron. https://questions-statements.parliament.uk/written-questions/detail/2025-06-23/HL8690
A question on #BattenDisease #MedicalTreatments tabled by Lord Botham on 23-06-2025 has been answered by Baroness Merron. https://questions-statements.parliament.uk/written-questions/detail/2025-06-23/HL8691
July 1, 2025 at 3:20 PM
A question on #BattenDisease #MedicalTreatments tabled by Lord Botham on 23-06-2025 has been answered by Baroness Merron. https://questions-statements.parliament.uk/written-questions/detail/2025-06-23/HL8691
A question on #BattenDisease #CerliponaseAlfa tabled by Lord Botham on 23-06-2025 has been answered by Baroness Merron. https://questions-statements.parliament.uk/written-questions/detail/2025-06-23/HL8693
July 1, 2025 at 3:20 PM
A question on #BattenDisease #CerliponaseAlfa tabled by Lord Botham on 23-06-2025 has been answered by Baroness Merron. https://questions-statements.parliament.uk/written-questions/detail/2025-06-23/HL8693
A question on #BattenDisease #CerliponaseAlfa tabled by Lord Botham on 23-06-2025 has been answered by Baroness Merron. https://questions-statements.parliament.uk/written-questions/detail/2025-06-23/HL8688
July 1, 2025 at 3:20 PM
A question on #BattenDisease #CerliponaseAlfa tabled by Lord Botham on 23-06-2025 has been answered by Baroness Merron. https://questions-statements.parliament.uk/written-questions/detail/2025-06-23/HL8688
A question on #BattenDisease #CerliponaseAlfa tabled by Lord Botham on 23-06-2025 has been answered by Baroness Merron. https://questions-statements.parliament.uk/written-questions/detail/2025-06-23/HL8692
July 1, 2025 at 3:20 PM
A question on #BattenDisease #CerliponaseAlfa tabled by Lord Botham on 23-06-2025 has been answered by Baroness Merron. https://questions-statements.parliament.uk/written-questions/detail/2025-06-23/HL8692
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-...
June 9, 2025 at 1:36 PM
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
A decade without this beautiful girl. I miss her so freaking much.
I love you, Mabel.
And I hate batten disease!!!!
#Yellow #BattenDisease #RareGenetics
May 30, 2025 at 1:10 AM
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
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.
I hate #BattenDisease so very much.
May 12, 2025 at 11:37 PM
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.
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.
My life advice: Cherish life. Oh, and fund rare disease research.
If you’re over 30, quote this with some life advice 🤌🏼
May 10, 2025 at 7:07 PM
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.
My life advice: Cherish life. Oh, and fund rare disease research.
Need to figure out how to get #BattenDisease research on his radar ASAP.
Bill Gates announced new plans to distribute “virtually all” of his wealth — around $200 billion, he estimates — within the next 20 years.
Bill Gates announces plan to give ‘virtually all’ his money away and end the Gates Foundation in 20 years | CNN Business
When Bill and Melinda French Gates created the Gates Foundation in 2000, they had planned for the organization to continue its work, using their remaining fortune, for several decades after they died....
www.cnn.com
May 8, 2025 at 1:31 PM
Need to figure out how to get #BattenDisease research on his radar ASAP.
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!
April 29, 2025 at 12:57 PM
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!
One of the many, many unexpected aspects of living in a #BattenDisease household!
There’s still plenty of time between now and Memorial Day, but the mattress and bedding sales are already underway. www.wired.com/story/best-e...
Best Early Memorial Day Mattress Deals
There’s still plenty of time between now and Memorial Day, but the mattress and bedding sales are already underway.
www.wired.com
April 26, 2025 at 10:22 AM
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!
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
Delighted to have Tom Wishart from @roslininstitute.bsky.social (Nottingham Trent) discuss the ‘Utility of Large Animal Models for Biomarker Identification’
#neuroday2025 #biomarkers #research
#neuroday2025 #biomarkers #research
April 25, 2025 at 5:45 AM
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
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...
#BattenResearch #BattenDisease #RareDisease #Science #Research #Clinical #Grant
www.facebook.com/share/v/19gh...
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April 4, 2025 at 9:24 AM
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...
#BattenResearch #BattenDisease #RareDisease #Science #Research #Clinical #Grant
www.facebook.com/share/v/19gh...