An Open-Label Extension Study of Patients with Late-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU02704 The objective of this extension study is to assess the long-term safety and efficacy of Myozyme treatment in patients with Late-Onset Pompe Disease who were previously treated under the placebo-contro...

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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 Prospective Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety and Efficacy of Mipomersen in Patients With Severe Hypercholesterolemia on a Maximally Tolerated Lipid-Lowering Regimen and Who are Not on Apheresis To compare the safety and efficacy of 26 weekly, subcutaneous injections of mipomersen (200 mg) against placebo in treating severely hypercholesterolemic patients who are on a maximally tolerated lipi...

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A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety and Efficacy of ISIS 301012 as Add-on Therapy in Homozygous Familial Hypercholesterolemia Subjects To evaluate the incremental LDL-C lowering efficacy of ISIS 301012 at Week 28 in homozygous familial hypercholesterolemia (HoFH) subjects treated for 26 weeks with ISIS 301012 added on to stable lipid...

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An Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of ISIS 301012 in Patients with Familial Hypercholesterolemia or Severe Hypercholesterolemia To evaluate the safety and efficacy of extended dosing with ISIS 301012 (mipomersen) in patients with familial hypercholesterolemia or severe hypercholesterolemia on concomitant lipid-lowering therapy

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Evaluation of Skeletal Myoblast Transplant for Treating Ischemic Heart Failure Phase 2 Study Myoblast Autologous Grafting in Ischemic Cardiomyopathy (MAGIC) Study The coprimary objectives of this study are to assess the recovery of contractility in akinetic myocardial segments that have received transplanted skeletal myoblasts as measured by echocardiogram, and...

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A Prospective, Randomized, Open-Label, Parallel-Group, Multicenter Study to Demonstrate the Superiority of Matrix-induced Autologous Chondrocyte Implantation (MACI® implant) versus Arthroscopic Microfracture for the Treatment of Symptomatic Articular Cartilage Defects of the Femoral Condyle including the Trochlea The objective of this study is to demonstrate superior efficacy and evaluate the safety of MACI implant compared with arthroscopic microfracture in the treatment of patients (aged 18 to 55 years old) ...

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