batten disease

Researchers find possible neuromarker for 'juvenile-onset' Batten disease

URMC - 08 Jan 2024
Researchers find possible neuromarker for 'juvenile-onset' Batten disease ...

Glycerophosphodiesters inhibit lysosomal phospholipid catabolism in Batten disease

ScienceDirect.com - 05 Mar 2024
Glycerophosphodiesters inhibit lysosomal phospholipid catabolism in Batten disease ...

A potential therapeutic target for juvenile Batten disease | Texas Children's

Texas Children’s - 03 Apr 2024
A potential therapeutic target for juvenile Batten disease | Texas Children's ...

Loss of the batten disease protein CLN3 leads to mis-trafficking of M6PR and defective autophagic-lysosomal reformation

Nature.com - 03 Jul 2023
Loss of the batten disease protein CLN3 leads to mis-trafficking of M6PR and defective autophagic-lysosomal reformation ...

Pigs provide a better model for Batten disease

Drug Discovery News - 16 Feb 2024
Pigs provide a better model for Batten disease ...

So long to 'The Long Sprint,' but not my fight against rare disease

Batten Disease News - 08 Feb 2024
So long to 'The Long Sprint,' but not my fight against rare disease ...

Advancement of potential gene therapy could offer new hope for children with Batten disease

News-Medical.Net - 28 Sep 2023
Advancement of potential gene therapy could offer new hope for children with Batten disease ...

Noah's Battle Against Batten: Baltimore Boy Crosses Skating Off His Bucket List

US Figure Skating - 20 Dec 2023
Noah's Battle Against Batten: Baltimore Boy Crosses Skating Off His Bucket List ...

'Deprioritized' gene therapy given as treatment for Batten disease

Batten Disease News - 04 Mar 2024
'Deprioritized' gene therapy given as treatment for Batten disease ...

Characterization of two human induced pluripotent stem cell lines derived from Batten disease patient fibroblasts ...

ScienceDirect.com - 19 Dec 2023
Characterization of two human induced pluripotent stem cell lines derived from Batten disease patient fibroblasts ... ...

Strategies proposed to treat juvenile Batten disease fear episodes

Batten Disease News - 02 Nov 2023
Strategies proposed to treat juvenile Batten disease fear episodes ...

CLN2 gene therapy shows safety, promising efficacy: Monkey study

Batten Disease News - 04 Jan 2024
CLN2 gene therapy shows safety, promising efficacy: Monkey study ...

Gene therapy programs for late infantile Batten disease halted

Batten Disease News - 07 Dec 2023
Gene therapy programs for late infantile Batten disease halted ...

Inhibiting Sortilin protein shows promise as Batten disease treatment

Batten Disease News - 05 Feb 2024
Inhibiting Sortilin protein shows promise as Batten disease treatment ...

RGX-181 cuts seizures by 80% in late infantile Batten disease...

Batten Disease News - 12 Oct 2023
RGX-181 cuts seizures by 80% in late infantile Batten disease... ...

How a late-infantile Batten disease diagnosis led to advocacy

Batten Disease News - 07 Feb 2024
How a late-infantile Batten disease diagnosis led to advocacy ...

Batten disease gene therapies to benefit from URMC funding boost |...

Batten Disease News - 07 Sep 2023
Batten disease gene therapies to benefit from URMC funding boost |... ...

A Mother's Dream to Rise Above – Cristina Vargas – Batten Disease...

Batten Disease News - 07 Feb 2024
A Mother's Dream to Rise Above – Cristina Vargas – Batten Disease... ...

Regenxbio advancing 2 gene therapies in pipeline for Batten |...

Batten Disease News - 24 Aug 2023
Regenxbio advancing 2 gene therapies in pipeline for Batten |... ...