This phase I/II first study of recombinant human GAA derived from CHO cells showed that rhGAA is capable of improving cardiac and skeletal muscle functions in infantile GSD-II patients. Further study will be needed to assess the overall potential of this therapy.

Four patients with cystic fibrosis (CF) were examined with combined hyperpolarized helium 3-enhanced and conventional proton magnetic resonance (MR) imaging. After inhalation of the polarized 3He gas, single breath-hold, gradient-echo images (resonant frequency of 3He) were obtained to depict lung ventilation. Conventional T2-weighted fast spin-echo (hydrogen) images were also obtained to depict morphologic abnormalities. 3He images were successfully and reproducibly generated that showed both morphologic abnormalities and, often more extensive, ventilation abnormalities. 3He MR imaging may provide a method for evaluating progression of pulmonary disease in patients with CF.

This phase I/II first study of recombinant human GAA derived from CHO cells showed that rhGAA is capable of improving cardiac and skeletal muscle functions in infantile GSD-II patients. Further study will be needed to assess the overall potential of this therapy.

Teens with cystic fibrosis or sickle cell disease took more potentially damaging health risks than might be expected, although the prevalence was lower than reported by their peers. Future longitudinal studies should examine the relationships between chronic illness, physical and psychosocial maturation, and risky behavior. Screening for psychosocial issues, including risky behaviors, should be incorporated into the routine health care of chronically ill teens.

No abstract