Evidence map›Paper›PMID 39744899›Full record

ArticleMuscle & nerve2025

Transitioning From Nusinersen to Risdiplam for Spinal Muscular Atrophy in Clinical Practice: A Single-Center Experience.

Can Ebru Bekircan-Kurt, Sharmada Subramanian, Shannon Chagat, Samuel J Mackenzie, Megan Iammarino, Natalie Reash, Carson Richardson, Chang-Yong Tsao, Garey Noritz, Courtney Gushue and 7 more

Abstract read
PubMed Full text at PMC Publisher
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Article in Muscle & nerve, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
7citing papers in PubMed, 1 pooled it
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

7 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. Review
  3. Review
  4. Review
  5. Article
  6. Review
  7. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

17 authors.

Can Ebru Bekircan-KurtDepartment of Neurology, School of Medicine, Hacettepe University, Ankara, Turkey.
Sharmada SubramanianCenter for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, Ohio, USA.
Shannon ChagatDepartment of Neurology, Neuromuscular Division, Nationwide Children's Hospital, Columbus, Ohio, USA.
Samuel J MackenzieDepartment of Neurology, Neuromuscular Division, University of Rochester Medical Center, Rochester, New York, USA.
Megan IammarinoCenter for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, Ohio, USA.
Natalie ReashCenter for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, Ohio, USA.ORCID https://orcid.org/0000-0002-0573-9504
Carson RichardsonBiostatistics Research Core, Ohio State University, Rochester, Ohio, USA.ORCID https://orcid.org/0009-0004-8710-1097
Chang-Yong TsaoDepartment of Neurology, Neuromuscular Division, Nationwide Children's Hospital, Columbus, Ohio, USA.
Garey NoritzDepartment of Pediatrics, Ohio State University, Wexner Medical Center, Columbus, Ohio, USA.
Courtney GushueDepartment of Pediatrics, Ohio State University, Wexner Medical Center, Columbus, Ohio, USA.
Kavitha KothaDepartment of Pediatrics, Ohio State University, Wexner Medical Center, Columbus, Ohio, USA.
Grace PaulDepartment of Pediatrics, Ohio State University, Wexner Medical Center, Columbus, Ohio, USA.
Richard ShellDepartment of Pediatrics, Ohio State University, Wexner Medical Center, Columbus, Ohio, USA.
Lindsay N AlfanoCenter for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, Ohio, USA.
Linda P LowesCenter for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, Ohio, USA.
Anne M ConnollyCenter for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, Ohio, USA.
Megan A WaldropCenter for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, Ohio, USA.ORCID https://orcid.org/0000-0001-9621-8319

Funding

AHRQ HHSAHRQ HHS K12 HS026393PEDSnet Scholars Training Program 5K12HS026393-03
6 · The paper itself

Abstract

backgroundNusinersen and risdiplam are U.S. Food and Drug Administration (FDA)-approved treatments for spinal muscular atrophy (SMA). No head-to-head clinical trials to assess efficacy exist. Observational studies are needed to determine if transitioning to risdiplam is safe and efficacious.

methodsThis retrospective study at Nationwide Children's Hospital included individuals with SMA treated with nusinersen who switched to risdiplam. Motor, pulmonary and bulbar function were assessed before and 2 years after nusinersen and risdiplam initiation.

resultsForty-four individuals were included: 11 with SMA type 1, 25 with SMA type 2 and 8 with SMA type 3. Motor function improved after initiation of nusinersen treatment with the most significant improvements seen in the first year. After transition to risdiplam, motor function remained largely stable. Need for noninvasive ventilation (NIV) overnight occurred in both groups. Cough peak flow significantly improved in the risdiplam group. Hospitalizations were the same in both groups. One individual in the nusinersen group gained the ability to take some food by mouth; two individuals in the risdiplam group achieved some oral feeding and two became exclusively orally fed.

conclusionsAs expected, motor function was most improved in treatment naïve individuals in the first year after nusinersen initiation. Over half of our study population had posterior spinal fusion surgery (57%) which significantly impacted motor and respiratory outcomes, though slightly less so in the risdiplam group. Overall, our data demonstrates that transitioning from nusinersen to risdiplam is associated with a favorable safety profile and stable motor outcomes.

Indexed as

Muscular Atrophy, SpinalOligonucleotidesSpinal Muscular Atrophies of ChildhoodAzo CompoundsChildChild, PreschoolDrug CombinationsFemaleHumansInfantMalePyrimidinesRetrospective StudiesTreatment OutcomeAzo CompoundsDrug CombinationsnusinersenOligonucleotidesPyrimidinesRisdiplamCHOPHFMSENIVnusinersenrisdiplamspinal muscular atrophy

Identifiers

PMID39744899
PMCPMC11799403

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LicenceCC BY-NC-ND
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Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.