Sampayo-Cordero, Miguel <br/><br/>
Agreement between the results of meta-analyses from case reports and from clinical studies regarding the efficacy of laronidase therapy in patients with mucopolysaccharidosis type I who initiated enzyme replacement therapy in adult age: An example of case reports meta-analyses as an useful tool for evidence-based medicine in rare diseases.  [electronic resource] 

 -  Molecular genetics and metabolism  02 2018 
 - 69-75 p.  digital 
<br/><br/> Publication Type: Journal Article; Review 
<br/><br/><label>ISSN: </label>1096-7206 
<br/><br/><label>Standard No.: </label>10.1016/j.ymgme.2018.01.002  doi 
<br/><br/><label>Subjects--Topical Terms: </label><br/>Adult<br/>Enzyme Replacement Therapy<br/>Evidence-Based Medicine<br/>Humans<br/>Iduronidase--therapeutic use<br/>Meta-Analysis as Topic<br/>Mucopolysaccharidosis I--therapy<br/>Rare Diseases--therapy