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5 Resultaat/Resultaten
Aanwerving proef
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; Lopend onderzoek
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; Terminated trial
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; Gefinancierd door een IRDiRC-lid = 
; Lid van een ERN = 
Nationale klinische studie(s)
USA
Maryland
BETHESDA
A Phase 1 Randomized, Placebo-Controlled, Double-Blind, Escalating Single-Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ManNAc in Subjects With GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)
National Human Genome Research Institute (NHGRI-NIH)
National Human Genome Research Institute
USA
Maryland
BETHESDA
Autologous Transplantation of Bone Marrow CD34+ Stem/Progenitor Cells After Addition of a Normal Human ADA cDNA by the EFS-ADA Lentiviral Vector for Adenosine Deaminase (ADA)-Deficient Severe Combined Immunodeficiency (SCID)
National Human Genome Research Institute (NHGRI-NIH)
National Human Genome Research Institute
USA
Maryland
BETHESDA
Phase 1 Dose Finding Trial of ARQ 092 in Children and Adults With Proteus Syndrome
National Human Genome Research Institute (NHGRI-NIH)
National Human Genome Research Institute
USA
Maryland
BETHESDA
Nonrandomized Open Label Pilot Study of Sirolimus Therapy for Segmental Overgrowth Caused by Somatic PI3K Activation
National Human Genome Research Institute (NHGRI-NIH)
National Human Genome Research Institute
USA
Maryland
BETHESDA