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Growth patterns in children with spinal muscular atrophy.

Ramona De AmicisGiovanni BaranelloAndrea FoppianiAlessandro LeoneAlberto BattezzatiGiorgio BedogniSimone RavellaEster GiaquintoChiara MastellaCaterina AgostoEnrico BertiniAdele D'AmicoMarina PedemonteClaudio BrunoJonathan C WellsMary FewtrellSimona Bertoli
Published in: Orphanet journal of rare diseases (2021)
These data on treatment-naïve patients point toward a better understanding of growth in SMA and could be useful to improve the clinical management and to assess the efficacy of the available and forthcoming therapies not only on motor function, but also on growth.
Keyphrases
  • end stage renal disease
  • ejection fraction
  • newly diagnosed
  • chronic kidney disease
  • young adults
  • prognostic factors
  • big data
  • combination therapy
  • data analysis