Noemi Coppola, Stefania Baldares, Andrea Blasi, Rosaria Bucci, Gianrico Spagnuolo, Michele Davide Mignogna, Stefania Leuci
Referral Patterns in Oral Medicine: A Retrospective Analysis of an Oral Medicine University Center in Southern Italy
International journal of environmental research and public health
advanced therapies offer unprecedented opportunities treating rare neurological disorders rnds children however health literacy perceptions understanding novel therapies need elucidation across rnd community study explored healthcare professionals carers perspectives advanced therapies childhoodonset rnds mixedmethodology crosssectional study 20 healthcare professionals clinicians genetic counsellors scientists 20 carers completed qualitative semistructured interviews customdesigned surveys carers undertook validated psychosocial questionnaires thematic quantitative data analysis followed participants described high positive interest advanced therapies low knowledge access reliable information substantial therapeutic gap therapeutic odyssey common rnds recognised five key themes unmet need urgency access ii seeking information iii access equity sustainability iv multidisciplinary integrated approach care support v difficult decisionmaking participants motivated intensify rnd clinical trial activity access advanced therapies however concerns around informed consent firstinhuman trials clinical trial procedures evident highrisk tolerance despite substantial uncertainties knowledge gaps rnds high mortality increased functional burdens alternative therapies consistently prioritised development advanced therapies however little consensus existed prioritisation treatment access study highlights need increase clinician health system readiness clinical translation advanced therapeutics rnds codevelopment use educational psychosocial resources support clinical decisionmaking set therapeutic expectations promotion equitable effective safe delivery advanced therapies essential participant insights psychosocial burden information need enhance delivery care formative study informing ongoing partnerships families including coproduction dissemination psychoeducational resources featuring voices hosted sydney childrens hospitals network website schn brainaid resources