Lysosomal Storage Diseases - 50 Studies Found
Recruiting |
: Pompe Telemedicine Developmental Study :
: 2016-06-22 |
Completed |
: Feasability and Interest of Screening for Infantile Pompe's Diseases at Birth : Pompe's Disease : 2016-09-06 : Other: observational (no intervention) |
Completed |
: Prevalence of Heterozygote Mothers for Pompe's Disease Among Mothers Having Delivered in French Guiana : Pompe's Disease : 2016-09-06 : Other: observational |
Not yet recruiting |
: VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Patients With Late-Onset Pompe Disease : Pompe Disease : 2016-09-08 :
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Recruiting |
: Investigating Pompe Prevalence in Neuromuscular Medicine Academic Practices : Pompe Disease : 2016-05-12 |
Recruiting |
: 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 |
Recruiting |
: Respiratory Muscle Training in L-Onset Pompe Disease (LOPD) : Glycogen Storage Disease Type II : 2016-06-13 :
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Recruiting |
: 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 :
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Recruiting |
: Identification of Tongue Involvement in Late-Onset Pompe Disease :
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Recruiting |
: Pompe Disease QMUS and EIM : Pompe Disease : 2016-04-11 :
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