Long-Term Follow-Up of Pediatric Patients with Dyskinetic Cerebral Palsy and Deep Brain Stimulation.

Background: Deep brain stimulation (DBS) has been increasingly used in the management of dyskinetic cerebral palsy (DCP). Data on long-term effects and the safety profile are rare.

Objectives: We assessed the efficacy and safety of pallidal DBS in pediatric patients with DCP.

Quality of Life After Deep Brain Stimulation of Pediatric Patients with Dyskinetic Cerebral Palsy: A Prospective, Single-Arm, Multicenter Study with a Subsequent Randomized Double-Blind Crossover (STIM-CP).

Background: Patients with dyskinetic cerebral palsy are often severely impaired with limited treatment options. The effects of deep brain stimulation (DBS) are less pronounced than those in inherited dystonia but can be associated with favorable quality of life outcomes even in patients without changes in dystonia severity.

Objective: The aim is to assess DBS effects in pediatric patients with pharmacorefractory dyskinetic cerebral palsy with focus on quality of life.

Differential Analysis of Bone Density in Children and Adolescents with Neuromuscular Disorders and Cerebral Palsy.

Determinants that affect bone density in disabled children and adolescents with neuromuscular disorders have not been differentially investigated well. We performed dual-energy X-ray absorptiometry of the lumbar spine in three groups (Duchenne muscular dystrophy, n = 16; other neuromuscular diseases, n = 11; and cerebral palsy, n = 18) providing height-age- and sex-adjusted z scores. Mobility was assessed by functional tests.

Somatropin treatment of spinal muscular atrophy: a placebo-controlled, double-blind crossover pilot study.

In preclinical studies growth hormone and its primary mediator IGF-1 have shown potential to increase muscle mass and strength. A single patient with spinal muscular atrophy reported benefit after compassionate use of growth hormone. Therefore we evaluated the efficacy and safety of growth hormone treatment for spinal muscular atrophy in a multicenter, randomised, double-blind, placebo-controlled, crossover pilot trial.