Table 1 Characteristics to guide family selection
From: Evidence of the impact of CLN2 and CLN3 Batten disease on families in the United Kingdom
Selection criteria | Exclusion criteria |
|---|---|
• Families of children with a confirmed diagnosis of CLN2/3 disease | • All other types of NCL |
• Parents or adult siblings who currently care, or had previously cared, for a child or children with CLN2/3 disease | • Members of the extended family who currently care, or had previously cared, for a child or children with CLN2/3 disease |
• Both atypical and typical CLN2/3 disease |  |
• All ages and ethnicities |  |
• All UK nations (Wales, England, Scotland and Northern Ireland) |  |
• Received or receiving cerliponase alfa; and did not receive cerliponase alfa (CLN2 only) • Any number of children in the family with CLN2/3 disease |  |
• Any number of children in the family with CLN2/3 disease |  |