Celeste Decker

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Pulmonary function is impaired in untreated mucopolysaccharidosis type VI (MPS VI). Pulmonary function was studied in patients during long-term enzyme replacement therapy (ERT) with recombinant human arylsulfatase B (rhASB; rhN-acetylgalactosamine 4-sulfatase). Pulmonary function tests prior to and for up to 240 weeks of weekly infusions of rhASB at 1 mg/kg(More)
BACKGROUND AND METHODS: Growth failure is characteristic of untreated mucopolysaccharidosis type VI (MPS VI: Maroteaux-Lamy syndrome). Growth was studied in fifty-six MPS VI patients (5 to 29 years old) prior to and for up to 240 weeks of weekly infusions of recombinant human arylsulfatase B (rhASB) at 1 mg/kg during Phase 1/2, Phase 2, Phase 3 or Phase 3(More)
BACKGROUND This study was conducted to identify potential biomarkers that could be used to evaluate disease progression and monitor responses to enzyme replacement therapy (ERT) in patients with mucopolysaccharidosis (MPS) IVA. METHODS Levels of 88 candidate biomarkers were compared in plasma samples from 50 healthy controls and 78 MPSIVA patients not(More)
Characteristic cardiac valve abnormalities and left ventricular hypertrophy are present in untreated patients with mucopolysaccharidosis type VI (MPS VI). Cardiac ultrasound was performed to investigate these findings in subjects during long-term enzyme replacement therapy (ERT) with recombinant human arylsulfatase B (rhASB, rhN-acetylgalactosamine(More)
OBJECTIVE To present long-term respiratory function outcomes from an open-label, multi-center, phase 3 extension study (MOR-005) of elosulfase alfa enzyme replacement therapy (ERT) in patients with Morquio A syndrome. METHODS In part 1 of MOR-005, patients initially randomized to ERT in the 24-week pivotal study (MOR-004) remained on their regimen (2.0(More)
OBJECTIVE To evaluate the efficacy and safety of two dose levels of galsulfase (Naglazyme®) in infants with MPS VI. STUDY DESIGN This was a phase 4, multicenter, multinational, open-label, two-dose level study. Subjects were randomized 1:1 to receive weekly infusions of 1.0 or 2.0 mg/kg of galsulfase for a minimum of 52 weeks. Progression of skeletal(More)
Efficacy and safety of elosulfase alfa enzyme replacement therapy (ERT) were assessed in an open-label, phase 2, multi-national study in Morquio A patients aged ≥5 years unable to walk ≥30 meters in the 6-min walk test. Patients received elosulfase alfa 2.0 mg/kg/week intravenously for 48 weeks. Efficacy measures were functional dexterity, pinch/grip(More)
* PAL-002 discontinuations: 1 due to AE, 1 lost to follow-up, 1 other (went to college); PAL-004 discontinuation: 1 withdrew consent. • All patients developed anti-PAL Ab responses and majority develop anti-PEG Ab responses. • Few patients tested positive for Neutralizing Antibodies (NAb). • No correlation between Ab positivity or titer level and HAE(More)
BACKGROUND To evaluate the outcome of surgical management of advanced squamous cell carcinoma (SCC) of the conjunctiva (American Joint Committee Cancer-classification >III) and the rate of recurrences after treatment during follow-up. Second, to investigate the incidence of orbital exenteration during follow-up. METHODS Thirty-eight cases with SCC >grade(More)
OBJECT In spite of the development of approximately 200 different shunt systems within the last 50 years, overdrainage and valve obstructions are still major problems in the current treatment of hydrocephalus. In 1994, a new gravitational valve with different opening pressures (depending upon the patient's posture) and a big contact area to CSF was(More)