Using Patient Reported Outcomes (PROs) to Evaluate Teriflunomide Treatment in Relapsing Multiple Sclerosis (RMS) Patients To describe efficacy, tolerability and convenience of teriflunomide treatment through the evaluation of PROs

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Safety, Efficacy, and Pharmacokinetics Study of Recombinant Human Acid Sphingomyelinase in Patients With Acid Sphingomyelinase Deficiency Sicurezza, Efficacia e Analisi di Farmacocinetica della sfingomielinasi acida ricombinante umana in pazienti con carenza di sfingomielinasi acida The primary objective is to evaluate the safety, efficacy, and pharmacokinetics of different doses of rhASM administered intravenously (IV) every 2 weeks for 52 weeks. L’obiettivo primario di...

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Efficacy, Safety and Pharmacokinetics of Teriflunomide in Pediatric Patients with Relapsing Forms of Multiple Sclerosis To assess the effect of teriflunomide in comparison to placebo on disease activity measured by time to first clinical relapse after randomization in children and adolescents 10 to 17 years of age with...

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A Study of the Safety and Efficacy of Two Different Regimens of Mipomersen in Patients With Familial Hypercholesterolemia and Inadequately Controlled Low-Density Lipoprotein Cholesterol Determine whether mipomersen (ISIS 301012) significantly reduces atherogenic lipid levels in patients with severe heterozygous familial hypercholesterolemia (severe HeFH), defined as low-density lipop...

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GZ/SAR402671 in Combination with Cerezyme in Adult Patients with Gaucher Disease Type 3 Part 1: -Evaluate central nervous system (CNS) biomarkers in Gaucher disease (GD) type 3 (GD3) that distinguish GD3 from Gaucher disease type 1 (GD1). -Screen adult GD3 patients who qualify for trea...

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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 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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NeoGAA Extension Study Long-term safety and pharmacokinetics (PK) of neoGAA

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Evaluation of efficacy and safety of vandetanib in patients with advanced papillary or poorly differentiated thyroid cancer To determine the efficacy (as assessed by progression free survival (PFS) ) of vandetanib when compared to placebo in patients with differentiated thyroid cancer that is either locally advanced or met...

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