In rare diseases, research based on registries and case studies is likely the best option, due to lack of patients, and case reports are often the primary evidence of the effectiveness of a new therapy or treatment [ 6 ]. Spearman rank correlation was used to evaluate relative agreement. An inclusive approach was used for low disease incidence. Outcomes not achieving this were classified as unacceptable evidence. Regarding publication bias, funnel plot tests cannot, be implemented when aggregating case reports [ 13 ]. There is evidence that case reports translate useful data collection in cases of rare phenomena, and contribute to the progress and dissemination of novel scientific discoveries three or more years earlier than clinical studies [ 11 ]. Long-term efficacy and safety of laronidase in the treatment of mucopolysaccharidosis I. These results confirm previous results observed in MPS-I adult patients.
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