10 results on '"Laforet, Pascal"'
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2. Improvements in the definition of biomarkers for Spinal Muscular Atrophy (SMA) type III and IV: a multimodal longitudinal study. (2321)
3. Long-Term Efficacy of Alglucosidase Alfa in Late-Onset Pompe Disease (P1.120)
4. Phase 1 Exploratory Efficacy of the Novel Enzyme Replacement Therapy NeoGAA in Treatment-Naïve and Alglucosidase Alfa-Treated Late-Onset Pompe Disease Patients (S38.006)
5. Phase 1 Safety and Pharmacokinetics of the Novel Enzyme Replacement Therapy neoGAA in Treatment-Naïve and Alglucosidase Alfa-Treated Late-Onset Pompe Disease Patients (S38.005)
6. Phase 1 Exploratory Efficacy of the Novel Enzyme Replacement Therapy NeoGAA in Treatment-Naïve and Alglucosidase Alfa-Treated Late-Onset Pompe Disease Patients (I4.011)
7. Asymptomatic Pompe Disease: A Study of 6 Patients. (P3.296)
8. A Diagnostic Flowchart For POLG Related Diseases Based On Signs Sensitivity And Specificity (P3.299)
9. Limb-Girdle Muscular Dystrophy Type 2E: Clinical, Genetic and Histopathological Features of 27 European Patients (P07.031)
10. Life-Threatening Lactic Acidosis Occuring in Adults with Rare Mutations of mtDNA: About Three Cases (P07.022)
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