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Prevalence of avoidant/restrictive food intake disorder in children and adolescents with rare diseases.
Blueprint for clinical N-of-1 strategies with off-label precision treatments in monogenic epilepsies.
The economic impact of caregiving for individuals with Angelman syndrome in the United States: results from a caregiver survey.
Association and causal impact of TERT genetic variants on peripheral blood leukocyte telomere length and cerebral small vessel disease risk in a Chinese Han population: a mendelian randomization analysis.
Lymphangioleiomyomatosis in patients with tuberous sclerosis: a national centre audit.