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Is treatment with oral risdiplam effective and well-tolerated for infants with spinal muscular atrophy type 1?

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  1. Sergott RC, Amorelli GM, Baranello G, Barreau E, Beres S, Kane S, et al. FIREFISH, SUNFISH, JEWELFISH Working Groups. Risdiplam treatment has not led to retinal toxicity in patients with spinal muscular atrophy. Ann Clin Transl Neurol. 2021;8:54–65. Epub 2020 Nov 24

    Article  CAS  Google Scholar 

  2. Szollosi A, Donkin C. Arrested theory development: the misguided distinction between exploratory and confirmatory research. Perspect Psychol Sci. 2021;16:717–24. Epub 2021 Feb 16

    Article  Google Scholar 

  3. Baranello G, Darras BT, Day JW, Deconinck N, Klein A, Masson R, et al. Risdiplam in type 1 spinal muscular atrophy. N Engl J Med. 2021;384:915–23. FIREFISH Working GroupEpub 2021 Feb 24

    Article  CAS  Google Scholar 

  4. Darras BT, Masson R, Mazurkiewicz-Bełdzińska M, Rose K, Xiong H, Zanoteli E, et al. Risdiplam-treated infants with type 1 spinal muscular atrophy versus historical controls. N Engl J Med. 2021;385:427–35. FIREFISH Working Group

    Article  CAS  Google Scholar 

  5. Pallmann P, Bedding AW, Choodari-Oskooei B, Dimairo M, Flight L, Hampson LV, et al. Adaptive designs in clinical trials: why use them, and how to run and report them. BMC Med. 2018;16:29.

    Article  Google Scholar 

  6. Lash TL, Vandenbroucke JP. Should preregistration of epidemiologic study protocols become compulsory? Reflections and a counterproposal. Epidemiology. 2012;23:184–8.

    Article  Google Scholar 

  7. Harvey LA. Trial registration: a pre-requisite for publication and an ethical responsibility. Spinal Cord. 2017;55:329–30.

    Article  CAS  Google Scholar 

  8. Vandenbroucke JP. Preregistration of epidemiologic studies: an ill-founded mix of ideas. Epidemiology. 2010;21:619–20.

    Article  Google Scholar 

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The Journal Club is a collaboration between the American Academy of Pediatrics – Section on Neonatal Perinatal Medicine and the International Society for Evidence-based Neonatology (

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All authors contributed equally towards initial conception and design, data analysis and interpretation, and manuscript composition and final approval.

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Correspondence to Sheema Gaffar.

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Gaffar, S., Cayabyab, R. & Ramanathan, R. Is treatment with oral risdiplam effective and well-tolerated for infants with spinal muscular atrophy type 1?. J Perinatol 42, 689–691 (2022).

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