Lysosomal Storage Diseases - 27 Studies Found

Completed : Long-term Outcome in Late-onset Pompe Disease Treated Beyond 36 Months (ATBIG-Pompe-Study)
: Pompe Disease
: 2016-06-20
: Drug: glucosidase alfa Long-term use in an observational study of licenced drug
Completed : 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
: Glycogen Storage Disease Type II-Pompe's Disease
: 2016-05-23
:
  • Drug: GZ402666

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

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

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

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