The NCS-LSD cohort study: a description of the methods and analyses used to assess the long-term effectiveness of enzyme replacement therapy and substrate reduction therapy in patients with lysosomal storage disorders

✍ Scribed by Henley, W. E.; Anderson, L. J.; Wyatt, K. M.; Nikolaou, V.; Anderson, R.; Logan, S.

• Book ID: 121554018
• Publisher: Springer
• Year: 2014
• Tongue: English
• Weight: 221 KB
• Volume: 37
• Category: Article
• ISSN: 0141-8955
• DOI: 10.1007/s10545-014-9679-6