Counteracting learned non-use in chronic stroke patients with reinforcement-induced movement therapy.

Background: After stroke, patients who suffer from hemiparesis tend to suppress the use of the affected extremity, a condition called learned non-use. Consequently, the lack of training may lead to the progressive deterioration of motor function. Although Constraint-Induced Movement Therapies (CIMT) have shown to be effective in treating this condition, the method presents several limitations, and the high intensity of its protocols severely compromises its adherence.

Agalsidase alfa in pediatric patients with Fabry disease: a 6.5-year open-label follow-up study.

Background: Signs and symptoms of the X-linked disorder, Fabry disease (FD), can occur early during childhood with heterogeneous clinical manifestations including potential cardiac and renal dysfunction. Several studies support the efficacy of the enzyme replacement therapy (ERT) agalsidase alfa, in adults with FD, though published data on the long-term safety and efficacy of agalsidase alfa in children are limited. As early treatment with ERT has the potential to reduce complications arising from disease progression, children in particular could benefit.

Four-year prospective clinical trial of agalsidase alfa in children with Fabry disease.

Objectives: To investigate a 4-year prospective clinical trial of agalsidase alfa in children with Fabry disease, an X-linked metabolic disorder caused by a deficiency of the lysosomal enzyme alpha-galactosidase A.

Study Design: Seventeen (16 boys, 1 girl; age range, 7.3 to 18.

High-dose cyclophosphamide treatment for refractory severe aplastic anemia in children.

Objective: To determine if high-dose cyclophosphamide is an effective therapy for children with refractory severe aplastic anemia (SAA).

Background: SAA is an illness characterized by the depletion of hematopoietic precursors associated with life-threatening complications. Hematopoietic stem cell transplant (HSCT) is the treatment of choice if a human leukocyte antigen (HLA)-related donor is available.

Unrelated bone marrow transplant for congenital amegakaryocytic thrombocytopenia: report of two cases and review of the literature.

CAMT is a very rare cause of thrombocytopenia in infants. Most of the patients will progress to marrow failure. Allogeneic stem cell transplant remains the only curative therapy.