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Lysosomal Storage Diseases - 27 Studies Found

Estado Estudiar
Completed Nombre del estudio: Long-term Outcome in Late-onset Pompe Disease Treated Beyond 36 Months (ATBIG-Pompe-Study)
Condición: Pompe Disease
Fecha: 2016-06-20
Intervenciones: Drug: glucosidase alfa Long-term use in an observational study of licenced drug
Completed Nombre del estudio: Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies neoGAA and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease
Condición: Glycogen Storage Disease Type II-Pompe's Disease
Fecha: 2016-05-23
Intervenciones:
  • Drug: GZ402666

Completed Nombre del estudio: Identification of Tongue Involvement in Late-Onset Pompe Disease
Condición:
  • Myopathy
  • Neuropathy
  • Glycogen Storage Disease Type II (Late
    Fecha: 2016-04-22
    Intervenciones: Other: Observational study The following exams will be done in all cohorts: tongue manual muscle testing
Enrolling by invitation Nombre del estudio: An MRI Study on Muscular Diseases -Pompe Disease and Dystrophia Myotonica-
Condición:
  • Glycogen Storage Disease Type 2
  • Dystrophia Myotonica

Fecha: 2015-12-08
Active, not recruiting Nombre del estudio: First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221
Condición: Pompe Disease
Fecha: 2016-01-26
Intervenciones:
  • Drug: ATB200
  • Drug: AT2221

Completed Nombre del estudio: Safety and Effectiveness of Resistance Exercise Training in Patients With Pompe Disease.
Condición:
  • Pompe Disease
  • Muscle Weakness

Fecha: 2015-12-10
Intervenciones: Other: Resistance Exercise Training Resistance Exercise Tr
Completed Nombre del estudio: Evaluation of Salbutamol as an Adjuvant Therapy for Pompe Disease
Condición: Pompe Disease
Fecha: 2015-03-22
Intervenciones: Drug: Salbutamol
  • Albuterol
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