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Updated: Jul 3, 2025
High Content Screening in Neurodegenerative Diseases
Published on: January 6, 2012
The Psychiatric Care of Children and Young Adults With Neurodegenerative Diseases
Aaron J Hauptman1, Erika F Augustine1, Heather B Brown1
1Kennedy Krieger Institute, Baltimore, Maryland; Johns Hopkins University School of Medicine, Baltimore, Maryland.
Insights
Pediatric neurodegenerative disorders (PNDs) cause progressive neurological decline and early death in children. These rare but devastating conditions present complex neuropsychiatric symptoms, impacting patients and families immensely.
Area of Science:
- Neurology
- Pediatrics
- Neuroscience
Background:
- Pediatric neurodegenerative disorders (PNDs) encompass over 70 rare conditions, affecting approximately 0.1 in 1,000 live births.
- These disorders lead to progressive neurological dysfunction, severe medical issues, and premature mortality in children and young adults.
- Examples include juvenile neuronal ceroid lipofuscinosis (CLN3 disease/Batten disease) and juvenile Huntington disease.
Discussion:
- PNDs manifest with complex neuropsychiatric symptoms, including cognitive decline, behavioral changes (aggression, self-injury), mood disorders, sensory disturbances, and psychosis.
- Symptom presentation is dynamic, evolving with disease progression and significantly impacting patient quality of life.
- The profound effects on affected children and their families result in substantial caregiver burden.
Key Insights:
- PNDs represent a significant challenge in pediatric neurology and neuropsychiatry due to their progressive nature and early mortality.
- The wide spectrum of neuropsychiatric symptoms requires specialized, multidisciplinary care approaches.
- Understanding the evolving nature of symptoms is crucial for effective management and support.
Outlook:
- Further research into the pathophysiology and treatment of PNDs is essential.
- Development of specialized pediatric neuropsychiatric and neuropalliative care clinics can improve patient and family outcomes.
- Enhanced diagnostic and therapeutic strategies are needed to address the unmet needs of children with PNDs.
Abstract:
Pediatric neurodegenerative disorders (PNDs), such as juvenile neuronal ceroid lipofuscinosis (CLN3 disease, also called Batten disease) and juvenile Huntington disease, are devastating conditions that result in progressive neurological dysfunction and profound medical comorbidities leading to early mortality in children and young adults.1 There are more than 70 PNDs, with a combined estimated prevalence of ∼0.1 in 1,000 live births.2,3 Individuals with PNDs commonly experience complex neuropsychiatric manifestations such as neurocognitive symptoms (dementia), irritability, aggression and self-injury, mood disorders, sensory alterations, and psychosis. Symptoms are dynamic, changing with illness progression, and evolve over time. Effects on patients and families can be devastating, and caregiver burden is enormous.4 We are a group of colleagues with backgrounds in pediatric neuropsychiatry, pediatric neuropalliative care, and pediatric neurology who care for patients together in specialized clinics.
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