#CLN2

µOrganoLab

@microorganolab.bsky.social

7 months ago

🚨 #PublicationAlert
Gene therapy for pediatric vision loss — powered by human retina models!
Our novel #RetinaOnChip mimics the human #Retina using #Organoids to model #CLN2 disease and test #GeneTherapy — now in a first-in-human trial!
📖 DOI: doi.org/10.1016/j.xc...

Edwin G. :mapleleafroundel:

@edwing.mstdn.moimeme.ca

7 months ago

British Columbia :flagbc: reinstated special coverage for a $1M/year medication against CLN2.

Me: It feels like the correct result.

www.cbc.ca/news/canada/british-colu...
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La Colombie-Britannique :flagbc: a rétabli la couverture […]

The Zebra Alliance Community

@thezebraalliance.bsky.social

8 months ago

'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

Redenlab Inc.

@redenlab.bsky.social

11 months ago

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