Enzyme replacement therapy in pediatric patients with Gaucher disease: what should we use as maintenance dosage?

Introduction: No consensus exists on the minimal dose of enzyme replacement therapy (ERT) effective to maintain therapeutic goals in pediatric Gaucher patients.

Objective: Evaluate the efficacy of low dosage ERT to maintain treatment goals.

Results: Six patients had a maintenance dose of 30-35U/kg/month.

Deflazacort use in Duchenne muscular dystrophy: an 8-year follow-up.

Data reported here were collected over an 8-year period for 79 Duchenne muscular dystrophy patients, 37 of whom were treated with deflazacort. Mean length of treatment was 66 months. Treated boys stopped walking at 11.

Effects of compressive vests on pulmonary function of infants with thoracic burn scars.

Objective: To prospectively evaluate the effects of compressive vests on the pulmonary function of infants with thoracic burn scars.

Methods: Between April 2000 and October 2005, all infants aged 2 years or less and all those aged between 2 and 3 years if they had concomitant pulmonary pathology, who were in need of a compressive vest for the treatment of burn scars to the thorax, underwent comparative pulmonary function testing under sedation with the vest closed and then opened.

Results: Of the 23 infants who met the inclusion criteria, 19 had complete data.

Feeding management of children with severe cerebral palsy and eating impairment: an exploratory study.

Some children with cerebral palsy and severe feeding impairment experience pulmonary complications from aspiration and gastroesophageal reflux. This exploratory study examined whether pulmonary function would improve following one year of intervention with optimal positioning for feeding, control of gastroesophageal reflux and use of food textures that would minimize aspiration from swallowing. Two children showed a 28% and 45% improvement, respectively, in functional residual capacity.