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Involvement of patient organisations in research activities: actions taken and lessons learned in a clinical research study for osteogenesis imperfecta.
Safety and efficacy of omaveloxolone v/s placebo for the treatment of Friedreich's ataxia in patients aged more than 16 years: a systematic review.
First-line nivolumab plus ipilimumab for unresectable MPM in China: a cost-effectiveness analysis.
Development and content validation of a symptom assessment for eosinophilic gastritis and eosinophilic gastroenteritis in adults and adolescents.
The frequency of rare and monogenic diseases in pediatric organ transplant recipients in Italy.