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3. Biallelic variation in the choline and ethanolamine transporter FLVCR1 underlies a severe developmental disorder spectrum

5. Bi-allelic Loss of Human APC2, Encoding Adenomatous Polyposis Coli Protein 2, Leads to Lissencephaly, Subcortical Heterotopia, and Global Developmental Delay

6. Cell-based analysis of CAD variants identifies individuals likely to benefit from uridine therapy

8. Biallelic variation in the choline and ethanolamine transporter FLVCR1 underlies a pleiotropic disease spectrum from adult neurodegeneration to severe developmental disorders

9. De novo and biallelic DEAF1 variants cause a phenotypic spectrum

11. Standardization of zebrafish drug testing parameters for muscle diseases

12. COL4A1 mutations cause ocular dysgenesis, neuronal localization defects, and myopathy in mice and Walker-Warburg syndrome in humans.

19. Mutations in B3GALNT2 Cause Congenital Muscular Dystrophy and Hypoglycosylation of α-Dystroglycan

30. Novel mutation identification and copy number variant detection via exome sequencing in congenital muscular dystrophy

33. Cell-based analysis ofCADvariants identifies individuals likely to benefit from uridine therapy

34. Cell-based analysis of CAD variants identifies individuals likely to benefit from uridine therapy

35. Cell-based analysis of CAD variants identifies individuals likely to benefit from uridine therapy

37. Overlap of polymicrogyria, hydrocephalus, and Joubert syndrome in a family with novel truncating mutations in ADGRG1/GPR56 and KIAA0556

39. Variable disease severity in Saudi Arabian and Sudanese families with c.3924 + 2 T > C mutation of LAMA2

40. Mutations in INPP5K Cause a Form of Congenital Muscular Dystrophy Overlapping Marinesco-Sjögren Syndrome and Dystroglycanopathy

48. Cell-based analysis of CADvariants identifies individuals likely to benefit from uridine therapy

49. CC2D1A Regulates Human Intellectual and Social Function as well as NF-κB Signaling Homeostasis

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