Effects of exercise on Pompe disease Plain English Summary under review.

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A Multicenter, Multinational, Open-Label Study of the Effects of Aldurazyme(laronidase) Treatment on Lactation in Women with Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants 1. To determine whether Aldurazyme affects lactation in women with MPS I. 2. To determine whether α-L-iduronidase is present in the breast milk of mothers with MPS I who are being treated with Aldura...

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Long-term Observational Follow-up Study of a Multicenter, Randomized, Double-blind, Placebo-controlled, Comparative Trial of AMD3100 (240 µg/kg) plus G-CSF (10 µg/kg) Versus G-CSF (10 µg/kg) plus Placebo to Mobilize and Collect ≥ 6×106 CD34+ Cells/kg in Multiple Myeloma Patients for Autologous Transplantation The objective of this long-term observational study is to assess progression-free survival and overall survival of patients treated with at least 1 dose of study treatment (placebo or plerixafor) for ...

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Full title of the trial : A Single Centre, Open-Label, Bridging Study of the Safety, Pharmacokinetics and Efficacy of Recombinant Human Acid Alpha-Glucosidase (rhGAA) Treatment in Patients with Late-Onset Pompe Disease (Glycogen Storage Disease Type II) Study the Safety, Pharmacokinetics and Efficacy of Recombinant Human Acid Alpha-Glucosidase (rhGAA) Treatment in Patients with Late-Onset Pompe Disease (Glycogen Storage Disease Type II)

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A study to assess the renal function in young male patients with Fabry disease who have never received any specific treatment for this disease. Estudio para evaluar la función renal en varones jóvenes con enfermedad de Fabry los cuales no han recibido nunca ningún tratamiento específico To document renal function and other Fabry disease manifestations across age in treatment-naïve, young male patients with Fabry disease. Documentar la función renal y otras manifestaciones de la...

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A Multicenter, Randomized, Comparative, Patient-blinded Study to Evaluate the Safety and Efficacy of G-CSF Alone Versus AMD3100 (240 µg/kg) Added to a G-CSF Mobilization Regimen in Adult Patients with Non-Hodgkin’s Lymphoma (NHL), Hodgkin’s Disease (HD) or Multiple Myeloma (MM) Who Have Previously Failed Stem Cell Collections To determine if patients reach a target of >= 2x10E6 cells/kg within 2 days of apheresis in NHL, HD or MM patients who are documented poor mobilizers that have received a mobilization regimen of G-CSF...

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Phase IIIB-IV long term follow-up study for patients who participated in CAMMS03409 To evaluate long-term safety of alemtuzumab

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Efficacy and Safety of Vatelizumab in Patients Who Completed Treatment in Study DRI13839 To assess the long-term safety of vatelizumab in MS patients

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A study to evaluate muscle tissue, blood, urine and imaging assessments in patients with Late-Onset Pompe Disease treated with alglucosidase alfa The primary objective of this study is to evaluate glycogen clearance in muscle tissue samples collected pre and post alglucosidase alfa treatment in patients with late-onset Pompe disease.

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A long-term study of olipudase alfa in patients with acid sphingomyelinase deficiency The primary objective of this study is to obtain data regarding the safety of olipudase alfa in patients with acid sphingomyelinase deficiency (ASMD) who are exposed to long-term treatment with olipud...

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