TY  - JOUR
AU  - Sampayo-Cordero, Miguel
AU  - Miguel-Huguet, Bernat
AU  - Pardo-Mateos, Almudena
AU  - Malfettone, Andrea
AU  - Pérez-García, José
AU  - Llombart-Cussac, Antonio
AU  - Cortés, Javier
AU  - Moltó-Abad, Marc
AU  - Muñoz-Delgado, Cecilia
AU  - Pérez-Quintana, Marta
AU  - Pérez-López, Jordi
PY  - 2019
DO  - 10.1186/s13023-019-1202-6
UR  - http://hdl.handle.net/10668/15167
T2  - Orphanet journal of rare diseases
AB  - A preliminary exploratory study shows solid agreement between the results of case reports and clinical study meta-analyses in mucopolysaccharidosis Type I (MPS-I) adult patients. The aim of the present study is to confirm previous results in another...
LA  - en
KW  - Case reports
KW  - Clinical studies
KW  - Enzyme replacement therapy
KW  - Evidence-based medicine
KW  - Meta-analysis
KW  - Mucopolysaccharidosis type II
KW  - Systematic review
KW  - Evidence-Based Medicine
KW  - Humans
KW  - Iduronate Sulfatase
KW  - Mucopolysaccharidosis II
KW  - Rare Diseases
TI  - Agreement between results of meta-analyses from case reports and clinical studies, regarding efficacy and safety of idursulfase therapy in patients with mucopolysaccharidosis type II (MPS-II). A new tool for evidence-based medicine in rare diseases.
TY  - research article
VL  - 14
ER  -