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5 results on '"Leypold, Christine"'

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1. Efficacy and safety of gene therapy with onasemnogene abeparvovec in children with spinal muscular atrophy in the D-A-CH-region: a population-based observational study

2. Improved upper limb function in non-ambulant children with SMA type 2 and 3 during nusinersen treatment: a prospective 3-years SMArtCARE registry study

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3. Clinical and genetic characteristics of late-onset Huntington's disease

4. Reduced Cancer Incidence in Huntington's Disease: Analysis in the Registry Study

5. Clinical manifestations of intermediate allele carriers in Huntington disease