TY  - JOUR
AU  - Pintos-Morell, Guillem
AU  - Blasco-Alonso, Javier
AU  - Couce, María L
AU  - Gutiérrez-Solana, Luís G
AU  - Guillén-Navarro, Encarna
AU  - O'Callaghan, Mar
AU  - Del Toro, Mireia
PY  - 2018
DO  - 10.1016/j.ymgmr.2018.03.009
SN  - 2214-4269
UR  - https://hdl.handle.net/10668/27103
T2  - Molecular genetics and metabolism reports
AB  - There is a growing interest in evaluating the effectiveness of enzyme replacement therapy (ERT) with elosulfase alfa in patients with mucopolysaccharidosis type IVA (MPS-IVA) under real-world conditions. We present the experience of seven pediatric...
LA  - en
KW  - Elosulfase alfa
KW  - Lysosomal storage disorder
KW  - MPS IVA
KW  - Morquio A
KW  - Quality of life
KW  - Urinary GAGs
TI  - Elosulfase alfa for mucopolysaccharidosis type IVA: Real-world experience in 7 patients from the Spanish Morquio-A early access program.
TY  - research article
VL  - 15
ER  -