Morquio A syndrome and effect of enzyme replacement therapy in different age groups of Turkish patients: a case series.
Sebile KılavuzSibel BasaranDeniz KorFatma Derya BulutSevcan ErdemHüseyin Tuğsan BallıMuhammed DağkıranSerdar CeylanerHalise Neslihan Onenli MunganPublished in: Orphanet journal of rare diseases (2021)
Overall, this case series with Morquio A patients confirms clinical trial data showing long-term stabilization of endurance after treatment initiation across ages and suggest that very early initiation of ERT optimizes growth outcomes.
Keyphrases
- end stage renal disease
- clinical trial
- replacement therapy
- chronic kidney disease
- ejection fraction
- newly diagnosed
- peritoneal dialysis
- prognostic factors
- type diabetes
- metabolic syndrome
- patient reported outcomes
- smoking cessation
- body composition
- electronic health record
- case report
- insulin resistance
- study protocol
- high intensity