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1. The multi-faceted nature of 15 CFTR exonic variations: Impact on their functional classification and perspectives for therapy

4. The multi-faceted nature of 15 CFTR exonic variations: Impact on their functional classification and perspectives for therapy

6. Prevalence and management of gastrointestinal manifestations in Silver–Russell syndrome

9. Implication of the SH3TC2 gene in Charcot-Marie-Tooth disease associated with deafness and/or scoliosis: Illustration with four new pathogenic variants

11. WS21.3 Overview of shared benefits from the 6-year long collaboration between the French Cystic Fibrosis Registry and the CFTR-France genetics database

15. The very low penetrance of cystic fibrosis for the R117H mutation: a reappraisal for genetic counselling and newborn screening

18. Age and origin of major Smith-Lemli-Opitz syndrome (SLOS) mutations in European populations

23. P017 Update of CFTR-France: toward a more relevant dataset for predicting the impact of rare CFTR variants

26. WS17.1 The multi-faceted nature of CFTR exonic mutations: impact on their functional classification

27. Efficiency of next generation sequencing of a large panel of genes for diagnosis of children with myopathies and muscular dystrophies, especially for early and/or typical cases

28. WS15.2 Massive parallel sequencing of the CFTR gene: a collaborative validation in diagnostic practice highlights strengths and limitations

29. WS15.1 CysMA, a new tool for the interpretation of rare CFTR missense variants

34. Efficiency of targeted NGS on myopathies and muscular dystrophy genes: Importance of an optimized strategy of capture, sequencing, bioinformatic analyses and multidisciplinary approach for variants detection and interpretation

35. 4 Valuable collaboration between a molecular CFTR database and a national CF registry: the French experience

41. Medical, psychological and social features in a large cohort of adults with Prader–Willi syndrome: experience from a dedicated centre in France

44. A molecular approach of dominance in hypophosphatasia

45. P.245 - Efficiency of targeted NGS on myopathies and muscular dystrophy genes: Importance of an optimized strategy of capture, sequencing, bioinformatic analyses and multidisciplinary approach for variants detection and interpretation

46. Renal insufficiency, a frequent complication with age in oral-facial-digital syndrome type I

49. WS8.5 Help for the interpretation of unclassified variants: example of the UMD-CFTR-France Locus Specific Database

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