Gene therapy benefits in a small cohort of symptomatic 5q spinal muscular atrophy patients and real world pediatric outcomes

Andresa R.B.V. Santos, Lisiane S. Ferreira, Tamires S. Santos, Juliana F. Mazzeu, Ney C.A. Boa Sorte

Open source

DOI
10.1016/j.jped.2026.101597
Published
2026-11
Container
Jornal de Pediatria
Publisher
Elsevier BV
Open access
unknown

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BibTeX

@article{allodium:10.1016/j.jped.2026.101597,
  title = {Gene therapy benefits in a small cohort of symptomatic 5q spinal muscular atrophy patients and real world pediatric outcomes},
  author = {Andresa R.B.V. Santos and Lisiane S. Ferreira and Tamires S. Santos and Juliana F. Mazzeu and Ney C.A. Boa Sorte},
  year = {2026},
  journal = {Jornal de Pediatria},
  doi = {10.1016/j.jped.2026.101597},
  url = {https://doi.org/10.1016/j.jped.2026.101597}
}

RIS

TY  - JOUR
TI  - Gene therapy benefits in a small cohort of symptomatic 5q spinal muscular atrophy patients and real world pediatric outcomes
AU  - Andresa R.B.V. Santos
AU  - Lisiane S. Ferreira
AU  - Tamires S. Santos
AU  - Juliana F. Mazzeu
AU  - Ney C.A. Boa Sorte
PY  - 2026
JO  - Jornal de Pediatria
DO  - 10.1016/j.jped.2026.101597
UR  - https://doi.org/10.1016/j.jped.2026.101597
ER  - 

APA

Santos, A. R., Ferreira, L. S., Santos, T. S., Mazzeu, J. F., & Sorte, N. C. B. (2026). Gene therapy benefits in a small cohort of symptomatic 5q spinal muscular atrophy patients and real world pediatric outcomes. Jornal de Pediatria. https://doi.org/10.1016/j.jped.2026.101597

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