BACKGROUND In Fabry disease, end-stage renal disease (ESRD) and severe neurologic and cardiac complications represent the leading causes of late morbidity and mortality. A comprehensive Italian nationwide survey study was conducted to explore changes in cardiac status and renal allograft function in Fabry patients on renal replacement therapy (RRT) and enzyme replacement therapy (ERT). METHOD...

BACKGROUND Late-onset Pompe disease is characterized by progressive skeletal myopathy followed by respiratory muscle weakness, typically leading to loss of ambulation and respiratory failure. In this population, enzyme replacement therapy (ERT) with alglucosidase alfa has been shown to stabilize respiratory function and improve mobility and muscle strength. Muscle pathology and glycogen clearan...

OBJECTIVES Determining the cost-effectiveness of enzyme replacement therapy (ERT) for the classical infantile form of Pompe disease (complete acid a-glucosidase deficiency-related) in two different settings: England and Colombia. Pompe disease is very rare (1:40,000 births incidence). METHODS A literature review was made and historic databases searched for National Health Service (NHS) reimbu...

Estrogen replacement therapy (ERT) is the main treatment postmenopausal osteoporosis. However, ERT causes serious side effects, such as cancers and thromboembolic problems. Labisia pumila var. alata (LPva) is a herb with potential as an alternative to ERT to prevent complications of osteoporosis, especially fragility fractures. This study was conducted to determine the effects of LPva on the bi...

AIM OF THE STUDY Postmenopausal osteoporosis is mainly treated with estrogen replacement therapy (ERT). However, ERT causes side effects, mainly breast cancer, uterine cancer and thromboembolic problems. Labisia pumila var. arata (LPva), a herb with phytoestrogenic effects has the potential to be used as an alternative agent to ERT. This study was conducted to determine the effects of LPva on b...

OBJECTIVE Activated protein C (APC) resistance not related to the factor V Leiden mutation is a risk factor for venous thrombosis. Oral estrogen replacement therapy (ERT) has been reported to induce APC resistance. Little is known about the effect of transdermal estrogen. METHODS AND RESULTS We enrolled 196 postmenopausal women who were randomly allocated to receive either 1 mg 17beta-estradi...

OBJECTIVE Because a categorical refusal of estrogen replacement therapy (ERT) from postmenopausal patients with a history of breast cancer is not based on any research evidence and may be more harmful than beneficial, we evaluated the safety and efficacy of ERT in these women. METHODS We recruited 131 patients who had been treated for breast cancer for a mean of 4.2 years (range 1 month to 20...

Since 2006 ERT is available in Europe and numerous studies were reported both in infantile and in juvenile adult patients that demonstrated variable efficacy (1, 2). We have followed infants treated with ERT either early in the first year or later. In one child with onset at birth, diagnosed in the third day of life we observed an excellent long-term clinical response on severe bradycardia and ...

Since 2006 ERT is available in Europe and numerous studies were reported both in infantile and in juvenile adult patients that demonstrated variable efficacy (1, 2). We have followed infants treated with ERT either early in the first year or later. In one child with onset at birth, diagnosed in the third day of life we observed an excellent long-term clinical response on severe bradycardia and ...