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A Randomized, Multicenter, Multinational, Phase 3B, Open-Label, Parallel-Group Study of Fabrazyme (agalsidase beta) in Treatment-Naive Male Pediatric Patients with Fabry Disease Without Severe Symptoms

The objectives of this open-label study are to evaluate the efficacy (GL-3 clearance), pharmacokinetics (PK), and safety parameters (including immunogenicity) for 2 alternative dose regimens of Fabrazyme (0.5 mg/kg every 2 weeks [q2w] and 1.0 mg/kg…

A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Gating or Residual Function Mutation (F/G and F/RF Genotypes)

Primary objective (Parts A and B):To evaluate the long-term safety and tolerability of VX-445/TEZ/IVA in subjects with CF who are heterozygous for the F508del mutation and a gating (F/G) or residual function (F/RF) mutation.Secondary objectives (…

An open-label study to evaluate the long-term safety and efficacy of evinacumab in patients with homozygous familial hypercholesterolemia

The primary objectives of the study are- To evaluate the long-term safety and tolerability of evinacumab 15 mg/kg intravenous (IV) administered every 4 weeks (Q4W) in patients with homozygous familial hypercholesterolemia (HoFH).- To evaluate the…

A Phase 3, Open-label, Rollover Study to Evaluate the Safety and Efficacy of
Long term Treatment With VX 661 in Combination With Ivacaftor in
Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous or
Heterozygous for the F508del CFTR Mutation

Main objective:Part ATo evaluate the long-term safety and tolerability of VX-661 in combination with ivacaftor in subjects with CF, homozygous or heterozygous for the F508del-CFTR mutation who are in the Treatment Cohort.Part B and Part CNot…

Randomized, controlled growth hormone study in children and adolescents with a Prader-Willi-like phenotype, followed by a two year open-label growth hormone study - effects on body composition, growth, psychosocial development and quality of life

To investigate the effects of GH treatment on body composition, psychosocial development, psychomotor development, growth, glucose metabolism, serum lipids, cognition, behaviour, bone mineral density, quality of life and safety parameters in…