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Rare Disease Research Laboratory » College of Medicine » University of Florida

A Phase 4, Open-Label, Prospective Study in Patients with Pompe Disease to Evaluate the Efficacy and Safety of Algulcosidase Alfa Produced at the 4000 L Scale. A Phase 4, Prospective, Multinational, Open-Label, Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment of Naive Patients with Infantile-Onset Pompe Disease. A Randomized, Double Blind, Placebo-Controlled, Phase 3 Trial of Tadalafil for Duchenne Muscular Dystrophy. Gene therapy treatment Glybera.

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Rare Disease Research Laboratory » College of Medicine » University of Florida | raredisease.powellcenter.med.ufl.edu Reviews
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A Phase 4, Open-Label, Prospective Study in Patients with Pompe Disease to Evaluate the Efficacy and Safety of Algulcosidase Alfa Produced at the 4000 L Scale. A Phase 4, Prospective, Multinational, Open-Label, Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment of Naive Patients with Infantile-Onset Pompe Disease. A Randomized, Double Blind, Placebo-Controlled, Phase 3 Trial of Tadalafil for Duchenne Muscular Dystrophy. Gene therapy treatment Glybera.
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Rare Disease Research Laboratory » College of Medicine » University of Florida | raredisease.powellcenter.med.ufl.edu Reviews

https://raredisease.powellcenter.med.ufl.edu

A Phase 4, Open-Label, Prospective Study in Patients with Pompe Disease to Evaluate the Efficacy and Safety of Algulcosidase Alfa Produced at the 4000 L Scale. A Phase 4, Prospective, Multinational, Open-Label, Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment of Naive Patients with Infantile-Onset Pompe Disease. A Randomized, Double Blind, Placebo-Controlled, Phase 3 Trial of Tadalafil for Duchenne Muscular Dystrophy. Gene therapy treatment Glybera.

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Sitemap » Powell Center for Rare Disease Research and Therapy » College of Medicine » University of Florida

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Meet the Clinical Team. Billing and Insurance FAQ. Powell Gene Therapy Center. Text-only version of this website. UF Health at Gainesville. UF Health Shands Hospital. UF Health Shands Cancer Hospital. Shands Emergency and Trauma. UF Health Shands Children's Hospital. UF Health Shands Rehab Hospital. UF Health Shands Psychiatric Hospital. Archer Family Health Care. Student Health Care Center. UF Health at Jacksonville. UF Health Proton Therapy Institute. College of Public Health and Health Professions.

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About Us » Powell Center for Rare Disease Research and Therapy » College of Medicine » University of Florida

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Meet the Clinical Team. Billing and Insurance FAQ. Powell Gene Therapy Center. Text-only version of this website. UF Health at Gainesville. UF Health Shands Hospital. UF Health Shands Cancer Hospital. Shands Emergency and Trauma. UF Health Shands Children's Hospital. UF Health Shands Rehab Hospital. UF Health Shands Psychiatric Hospital. Archer Family Health Care. Student Health Care Center. UF Health at Jacksonville. UF Health Proton Therapy Institute. College of Public Health and Health Professions.

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News » Powell Center for Rare Disease Research and Therapy » College of Medicine » University of Florida

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Meet the Clinical Team. Billing and Insurance FAQ. Powell Gene Therapy Center. Text-only version of this website. UF Health at Gainesville. UF Health Shands Hospital. UF Health Shands Cancer Hospital. Shands Emergency and Trauma. UF Health Shands Children's Hospital. UF Health Shands Rehab Hospital. UF Health Shands Psychiatric Hospital. Archer Family Health Care. Student Health Care Center. UF Health at Jacksonville. UF Health Proton Therapy Institute. College of Public Health and Health Professions.

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Support our Mission » Powell Center for Rare Disease Research and Therapy » College of Medicine » University of Florida

http://raredisease.powellcenter.med.ufl.edu/donate

Meet the Clinical Team. Billing and Insurance FAQ. Powell Gene Therapy Center. Text-only version of this website. UF Health at Gainesville. UF Health Shands Hospital. UF Health Shands Cancer Hospital. Shands Emergency and Trauma. UF Health Shands Children's Hospital. UF Health Shands Rehab Hospital. UF Health Shands Psychiatric Hospital. Archer Family Health Care. Student Health Care Center. UF Health at Jacksonville. UF Health Proton Therapy Institute. College of Public Health and Health Professions.

5

Clinical Trials » Powell Center for Rare Disease Research and Therapy » College of Medicine » University of Florida

http://raredisease.powellcenter.med.ufl.edu/clinical-trial

Meet the Clinical Team. Billing and Insurance FAQ. Powell Gene Therapy Center. Text-only version of this website. UF Health at Gainesville. UF Health Shands Hospital. UF Health Shands Cancer Hospital. Shands Emergency and Trauma. UF Health Shands Children's Hospital. UF Health Shands Rehab Hospital. UF Health Shands Psychiatric Hospital. Archer Family Health Care. Student Health Care Center. UF Health at Jacksonville. UF Health Proton Therapy Institute. College of Public Health and Health Professions.

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Rare Disease Research Laboratory » College of Medicine » University of Florida

A Phase 4, Open-Label, Prospective Study in Patients with Pompe Disease to Evaluate the Efficacy and Safety of Algulcosidase Alfa Produced at the 4000 L Scale. A Phase 4, Prospective, Multinational, Open-Label, Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment of Naive Patients with Infantile-Onset Pompe Disease. A Randomized, Double Blind, Placebo-Controlled, Phase 3 Trial of Tadalafil for Duchenne Muscular Dystrophy. Gene therapy treatment Glybera.

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