Early initiation of ambroxol treatment diminishes neurological manifestations of type 3 Gaucher disease: A long-term outcome of two siblings.

Gaucher disease type 3 (GD3) is a severely debilitating disorder characterized by multisystemic manifestations and neurodegeneration. Enzyme replacement therapy alleviates visceral signs and symptoms but has no effect on neurological features. Ambroxol has been suggested as an enzyme enhancement agent.

Prognostic value of cranial ultrasonography in comparison with magnetic resonance imaging in children with cerebral palsy: a population-based study.

The aim of this population-based study was to evaluate the characteristics of cerebral palsy (CP) in relation to the predominant pattern of the Magnetic Resonance Imaging Classification System (MRICS) that was analogously applied to the neonatal/early infant cranial ultrasound (CUS). The study included children born during the 2004-2007 period from the Croatian part (C28 RCP-HR) of the Surveillance of Cerebral Palsy in Europe (SCPE) CP register. Motor functions, accompanying impairments and brain MRI were evaluated in 227 children, 185 of which also had CUS.

Neurologic parameters in the perinatal period in children with neurodevelopmental disorders.

Unlabelled: Cerebral palsy (CP) is a term encompassing a group of nonprogressive, noncontagious conditions causing mild, moderate or severe disorders of neurodevelopment.

Objective: Objective of this study was to analyze the possible prenatal etiological factors for the emergence of neurodevelopmental disorders (NDs) and CP from the medical records of 100 children with neuromotor disabilities who were treated in Special Hospital for Children with Neuro-developmental and Movement Disorders, Goljak, Croatia.

Results: ND and CP were more often diagnosed in children with birth weight below 2500 g which was statistically proved at the level of significance reaching 0.