August 26, 2025 • 1 min
New research from the Lysosomal Disease Network (LDN). This summary is based on a paper published in the journal Pediatric Neurology on June 16, 2025, titled "Impact of CLN3 Disease on Child Quality of Life and Family Function."
Read the paper here.
Learn more about LDN.
Transcript:
New research from the Lysosomal Disease Network (LDN), a research group of the Rare Diseases Clinical Research Network.
Evaluating Health-Related Quality of Life and Family Function in CLN3 Disease.
This summary is based on a paper published in the journal Pediatric Neurology on June 16, 2025.
CLN3 disease, also known as Batten disease, is an inherited neurodegenerative disorder that typically starts in childhood. Because CLN3 disease is progressive and fatal, it is likely to affect the health-related quality of life (HRQOL) of both the child and the family.
In this study, researchers evaluated the HRQOL and family function in individuals with CLN3 disease and their families. The team analyzed data from 71 participants who completed assessments on the child HRQOL and family impact. In 21 of these participants, the team also analyzed Unified Batten Disease Rating Scale responses.
Results showed an association between worse child HRQOL and more severe disease symptoms, including physical impairment and functional capability. However, family impact severity was not associated with CLN3 disease symptom severities. While child HRQOL worsened over time, family impact did not significantly change over time. Authors note that these findings may provide important information for clinical care and trial design in CLN3 disease.
Read the paper here.
Learn more about LDN.
Transcript:
New research from the Lysosomal Disease Network (LDN), a research group of the Rare Diseases Clinical Research Network.
Evaluating Health-Related Quality of Life and Family Function in CLN3 Disease.
This summary is based on a paper published in the journal Pediatric Neurology on June 16, 2025.
CLN3 disease, also known as Batten disease, is an inherited neurodegenerative disorder that typically starts in childhood. Because CLN3 disease is progressive and fatal, it is likely to affect the health-related quality of life (HRQOL) of both the child and the family.
In this study, researchers evaluated the HRQOL and family function in individuals with CLN3 disease and their families. The team analyzed data from 71 participants who completed assessments on the child HRQOL and family impact. In 21 of these participants, the team also analyzed Unified Batten Disease Rating Scale responses.
Results showed an association between worse child HRQOL and more severe disease symptoms, including physical impairment and functional capability. However, family impact severity was not associated with CLN3 disease symptom severities. While child HRQOL worsened over time, family impact did not significantly change over time. Authors note that these findings may provide important information for clinical care and trial design in CLN3 disease.
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