Clinical Trials

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Clinical Trial
  • Status: Enrolling by invitation
  • Intervention Type: Other
  • Participants: 18
  • Start Date: July 15, 2019
A Long-Term Follow-up Study to Evaluate the Safety and Efficacy of Adeno Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Glucose-6-Phosphatase (G6Pase) in Adults With Glycogen Storage Disease Type Ia (GSDIa)
Clinical Trial
  • Status: Active, not recruiting
  • Phase: Phase 1/Phase 2
  • Intervention Type: Genetic
  • Participants: 12
  • Start Date: July 24, 2018
A Phase 1/2, Open-Label Safety and Dose-Finding Study of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Glucose-6- Phosphatase (G6Pase) in Adults With Glycogen Storage Disease Type Ia (GSDIa)
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Clinical Trial
  • Status: Recruiting
  • Intervention Type: Other
  • Participants: 200
  • Start Date: December 2015
Database for Information on Individuals Affected With Glycogen Brancher Deficiency, Also Known as Glycogen Storage Disease Type IV
Clinical Trial
  • Status: Recruiting
  • Phase: Phase 2
  • Intervention Type: Drug, Biological
  • Participants: 70
  • Start Date: April 2014
A Phase II Trial of HSCT for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Risk-Adjusted Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine
Clinical Trial
  • Status: Recruiting
  • Participants: 20000
  • Start Date: July 2010
Coordination of Rare Diseases at Sanford
Clinical Trial
  • Status: Active, not recruiting
  • Phase: Phase 4
  • Intervention Type: Biological
  • Participants: 30
  • Start Date: August 26, 2008
A Long-term Study to Evaluate Growth and Development Outcomes in Patients With Infantile-Onset Pompe Disease Who Are Receiving Alglucosidase Alfa.
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