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35 results on '"Ferlini, Alessandra"'

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2. Digital health and Clinical Patient Management System (CPMS) platform utility for data sharing of neuromuscular patients: the Italian EURO-NMD experience

3. AFG3L2 Biallelic Mutation: Clinical Heterogeneity in Two Italian Patients

4. Gain and loss of TASK3 channel function and its regulation by novel variation cause KCNK9 imprinting syndrome

6. Recommendations for whole genome sequencing in diagnostics for rare diseases

7. Improving diagnosis for rare diseases: the experience of the Italian undiagnosed Rare diseases network

10. Erratum: Corrigendum: Translation from a DMD exon 5 IRES results in a functional dystrophin isoform that attenuates dystrophinopathy in humans and mice

12. Genetic counseling for women referred for advanced maternal age: a telegenetic approach

13. Translation from a DMD exon 5 IRES results in a functional dystrophin isoform that attenuates dystrophinopathy in humans and mice

14. Patterns of late gadolinium enhancement in Duchenne muscular dystrophy carriers

15. Exome sequencing in a family with intellectual disability, early onset spasticity, and cerebellar atrophy detects a novel mutation in EXOSC3

16. Characterization of a rare case of Ullrich congenital muscular dystrophy due to truncating mutations within the COL6A1 gene C-Terminal domain: a case report

17. Genetic characterization in symptomatic female DMD carriers: lack of relationship between X-inactivation, transcriptional DMD allele balancing and phenotype

18. The absence of dystrophin brain isoform expression in healthy human heart ventricles explains the pathogenesis of 5' X-linked dilated cardiomyopathy

21. Usefulness and limitations of 99mTc-3,3-diphosphono-1,2-propanodicarboxylic acid scintigraphy in the aetiological diagnosis of amyloidotic cardiomyopathy

23. Custom CGH array profiling of copy number variations (CNVs) on chromosome 6p21.32 (HLA locus) in patients with venous malformations associated with multiple sclerosis

24. Identification of a deep intronic mutation in the COL6A2 gene by a novel custom oligonucleotide CGH array designed to explore allelic and genetic heterogeneity in collagen VI-related myopathies

26. A novel custom high density-comparative genomic hybridization array detects common rearrangements as well as deep intronic mutations in dystrophinopathies

27. Screening of mutations in the CFTR gene in 1195 couples entering assisted reproduction technique programs

29. Mutation analysis of the MECP2 gene in British and Italian Rett syndrome females

30. The role of immunocytochemistry and linkage analysis in the prenatal diagnosis of merosin-deficient congenital muscular dystrophy

32. Haplotype analysis of common transthyretin mutations

33. Parental origin and germline mosaicism of deletions and duplications of the dystrophin gene: a European study

34. Familial amyloidotic polyneuropathy: transthyretin (prealbumin) variants in kindreds of Italian origin

35. Identification of a deep intronic mutation in the COL6A2 gene by a novel custom oligonucleotide CGH array designed to explore allelic and genetic heterogeneity in collagen VI-related myopathies

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