About 39% of surveyed families used cannabis for children with a rare neurodegeneration causing severe dystonia

7 of 18 respondents (39%) had used cannabis for their child. Cannabis users were on more medications, more likely to have used opiates, and had greater physical impairment. Four children reported moderate or significant improvement in dystonia, 3 in pain, 4 in sleep, 3 in anxiety, and 2 in behavior. Adverse effects were mild (sadness, agitation, tiredness in 1 each).

Cross-sectional 37-item survey distributed to families of 44 children in a clinical registry of pantothenate kinase-associated neurodegeneration. 18 responses (41% response rate). Cannabis use, sources of information, symptom changes, and adverse effects collected.

Children with rare neurodegenerative diseases and severe dystonia often exhaust standard treatments. This survey reveals that families are turning to cannabis and reporting benefits, highlighting the need for controlled studies in pediatric movement disorders.

This study adds to growing evidence that families of children with severe, treatment-resistant neurological conditions are using cannabis, often based on information from other parents rather than medical providers.

Very small sample (18 respondents, 7 cannabis users). Survey response bias likely favoring families with strong opinions about cannabis. No objective outcome measures. No control group for comparison.