Evidence map›Paper›PMID 40153113›Full record

ArticleNeurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology2025

Patients on treatment with risdiplam in Italy: challenges in the interpretation of the real-world data.

Emilio Albamonte, Adrea Lizio, Giorgia Coratti, Lorenzo Maggi, Elena Pegoraro, Marika Pane, Sonia Messina, Riccardo Masson, Adele D'Amico, Enrico Bertini and 16 more

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In one paragraph

Article in Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers.

0numbers the graph read from it
0cells of the map it votes in
5citing papers in PubMed
–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

5 citing papers in PubMed.

  1. Review
  2. Article
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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

26 authors.

Emilio AlbamonteThe NEMO Center in Milan, Neurorehabilitation Unit, University of Milan, ASST Niguarda Hospital, Milan, Italy. emilio.albamonte@centrocliniconemo.it.ORCID http://orcid.org/0000-0002-8465-4460
Adrea LizioThe NEMO Center in Milan, Neurorehabilitation Unit, University of Milan, ASST Niguarda Hospital, Milan, Italy.
Giorgia CorattiPediatric Neuromuscular Unit, The NEMO Center in Rome, Rome, Italy.
Lorenzo MaggiNeuroimmunology and Neuromuscular Disease Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.
Elena PegoraroDepartment of Neurosciences, University of Padova, Padua, Italy.
Marika PanePediatric Neurology, Department of Woman and Child Health and Public Health, Università Cattolica del Sacro Cuore, Child Health Area, Rome, Italy.
Sonia MessinaDepartment of Clinical and Experimental Medicine, University of Messina, Messina, Italy.
Riccardo MassonNeuroimmunology and Neuromuscular Disease Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.
Adele D'AmicoUnit of Muscular and Neurodegenerative Disorders, Bambino Gesù Children's Hospital, Rome, Italy.
Enrico BertiniUnit of Muscular and Neurodegenerative Disorders, Bambino Gesù Children's Hospital, Rome, Italy.
Antonella PiniNeuromuscular Pediatric Unit, UOC Di Neuropsichiatria Dell'età Pediatrica, IRCCS Istituto Delle Scienze Neurologiche Di Bologna, Bologna, Italy.
Federica RicciNeuromuscular Center, AOU Città Della Salute E Della Scienza, University of Turin, Turin, Italy.
Tiziana MonginiDepartment of Neurosciences Rita Levi Montalcini, University of Turin, Turin, Italy.
Claudio BrunoCentre of Translational and Experimental Myology, Department of Neuroscience, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health-DINOGMI, IRCCS Istituto Giannina Gaslini, University of Genoa, Genoa, Italy.
Katia PatanellaThe NEMO Center in Rome, Adult Neuromuscular Unit, Rome, Italy.
Maria SframeliDepartment of Clinical and Experimental Medicine, University of Messina, Messina, Italy.
Claudia DosiNeuroimmunology and Neuromuscular Disease Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.
Silvia BonannoNeuroimmunology and Neuromuscular Disease Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.
Gaia ScarpiniNeuromuscular Pediatric Unit, UOC Di Neuropsichiatria Dell'età Pediatrica, IRCCS Istituto Delle Scienze Neurologiche Di Bologna, Bologna, Italy.
Noemi BrolattiCentre of Translational and Experimental Myology, Department of Neuroscience, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health-DINOGMI, IRCCS Istituto Giannina Gaslini, University of Genoa, Genoa, Italy.
Alice ZanoliniThe NEMO Center in Milan, Neurorehabilitation Unit, University of Milan, ASST Niguarda Hospital, Milan, Italy.
Chiara BravettiPediatric Neuromuscular Unit, The NEMO Center in Rome, Rome, Italy.
Maria Carmela PeraPediatric Neuromuscular Unit, The NEMO Center in Rome, Rome, Italy.
Eugenio Maria MercuriPediatric Neuromuscular Unit, The NEMO Center in Rome, Rome, Italy. emilio.albamonte@centrocliniconemo.it.
Valeria Ada SansoneThe NEMO Center in Milan, Neurorehabilitation Unit, University of Milan, ASST Niguarda Hospital, Milan, Italy. emilio.albamonte@centrocliniconemo.it.
ItaSMAc

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

aims(i) provide a snapshot from a large cohort of Italian patients with SMA on risdiplam in the real-world setting; (ii) identify any differences in the cohorts before and after commercial drug approval considering the different eligibility access criteria (iii) describe preliminary data on adherence to treatment and reasons for shifting from nusinersen to risdiplam.

methodsCharts from patients on risdiplam were retrospectively reviewed. Results were then compared between patients accessing the drug during an initial restricted compassionate use program (cohort 1) and those after commercial approval, with no restrictions (cohort 2). Side effects and adherence were recorded for both cohorts as well as data on shifters.

results283 patients (median age: 22 years) were included. Only a minority were walkers. Respiratory and bulbar comorbidities were more severe in cohort 1 (58% non sitters) than in cohort 2 (52% sitters). 35% and 46% of patients from cohorts 1 and 2 shifted from nusinersen to risdiplam respectively. Adherence and safety profile were good in both cohorts.

conclusionsThis is the largest cohort described so far providing insights on the characteristics of patients on risdiplam in the real world. The disability level and age were very different from those that had driven efficacy results in the trials. This may at least in part produce some evidence to account for the variable results reported so far in the realworld. Importantly, the safety profile was confirmed even in these more severely disabled and older patients compared to those in the trials.

Indexed as

OligonucleotidesAdolescentAdultChildChild, PreschoolCohort StudiesFemaleHumansInfantItalyMaleMiddle AgedRetrospective StudiesYoung AdultnusinersenOligonucleotidesAdherenceCompassionate use programRisdiplamSafetySpinal Muscular Atrophy

Identifiers

PMID40153113

What OpenQuestion holds

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Registered trials

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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.