Deficiency Diseases - 33 Studies Found

Completed : IMM 0212: Busulfan With Fludarabine and Antithymocyte Globulin as Preparative Therapy for Hematopoietic Stem Cell Transplant for the Treatment of Severe Congenital T-Cell Immunodeficiency
:
  • T-Cell Immune Deficiency Diseases
  • Severe Combined Immunodeficie
    : 2005-09-27
    : Drug: Busulfan, Fludarabine and ATG
Completed : International Collaborative Gaucher Group (ICGG) Gaucher Registry
:
  • Gaucher Disease
  • Cerebroside Lipidosis Syndrome
  • Glucocerebr
    : 2006-07-12
Completed : A Multicenter Study of the Efficacy of Cerezyme in Testing Skeletal Disease in Patients With Type I Gaucher Disease.
:
  • Gaucher Disease Type I
  • Cerebroside Lipidosis Syndrome
  • Cluc
    : 2006-08-15
    : Drug: Cerezyme (imiglucerase for injection)
Completed : Safety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher Disease
:
  • Gaucher Disease, Type 1
  • Cerebroside Lipidosis Syndrome
  • Glu
    : 2006-08-15
    : Drug: Cerezyme Cerezyme doses of 20-60U/kg every 2 weeks (Q2 Arm) or 40-120 U/kg every 4 weeks (Q4 Arm).
Completed : A Study of the Efficacy and Safety of Eliglustat Tartrate (Genz-112638) in Type 1 Gaucher Patients
:
  • Gaucher Disease, Type 1
  • Cerebroside Lipidosis Syndrome
  • Glu
    : 2006-07-27
    : Drug: Eliglustat tartrate Eliglustat (Genz-112638) capsule as single 50 mg dose on Day 1 then eliglustat
Active, not recruiting : Growth and Development Study of Alglucosidase Alfa.
:
  • Pompe Disease
  • Glycogen Storage Disease Type II (GSD-II)
  • Ac
    : 2007-06-13
    : Biological: alglucosidase alfa Intravenous (IV) infusion: 20mg/kg every 2 weeks
Approved for marketing : Alglucosidase Alfa Temporary Access Program
:
  • Glycogen Storage Disease Type II (GSD-II)
  • Pompe Disease (Late-Onset)
  • Pompe Disease (Late-onset)
  • Glycogen Storage Disease Type II (GSD-II)
  • Pompe Disease (Late-onset)
  • Glycogen Storage Disease Type II (GSD-II)
  • Pompe Disease (Late-onset)
  • Glycogen Storage Disease Type II (GSD-II)
  • Biological: alglucosidase alfa

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