Observational study in Journal of neuromuscular diseases, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.
0numbers the graph read from it
0cells of the map it votes in
2citing 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.
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
18 authors.
Lamiae GrimaldiClinical Research Unit, Assistance Publique - Hôpitaux de Paris University Paris-Saclay, Faculty of Medicine, University of Versailles Saint-Quentin en Yvelines, Inserm U1018 Team Anti-infective evasion and pharmacoepidemiology, Boulogne-Billancourt, France.ORCID 0000-0003-1952-5467
Rocio Garcia-UzquianoPediatric Neurology and ICU Department, Garches Reference Centre for Neuromuscular Diseases (NEIF for FILNEMUS; RPC for Euro-NMD ERN), AP-HP Paris Saclay University, Hôpital Raymond Poincaré (UVSQ), 104 boulevard Raymond Poincaré, 92380 Garches, France.ORCID 0009-0007-5402-4883
Marta Gomez-Garcia de la BandaPediatric Neurology and ICU Department, Garches Reference Centre for Neuromuscular Diseases (NEIF for FILNEMUS; RPC for Euro-NMD ERN), AP-HP Paris Saclay University, Hôpital Raymond Poincaré (UVSQ), 104 boulevard Raymond Poincaré, 92380 Garches, France.ORCID 0000-0002-2252-0694
Amal Oulhissane-OmarPediatric Neurology and ICU Department, Garches Reference Centre for Neuromuscular Diseases (NEIF for FILNEMUS; RPC for Euro-NMD ERN), AP-HP Paris Saclay University, Hôpital Raymond Poincaré (UVSQ), 104 boulevard Raymond Poincaré, 92380 Garches, France.
Celine TardNeurology and Movement Disorders Department, U1172, University Hospital Centre (CHU) de Lille, Reference Centre for Neuromuscular Diseases Nord Est Ile de France, Lille, France.ORCID 0000-0002-6654-8090
Pascale Saugier-VeberGenetics Department and Reference Centre for Developmental Disorders, Normandie Univ, UNIROUEN, CHU Rouen, Inserm U1245, FHU G4 Génomique, F-76000, Rouen, France.ORCID 0000-0002-8045-6432
Vincent LaugelPaediatrics Department, Strasbourg University Hospital, University of Strasbourg, Inserm UMRS 1112, Strasbourg, France.
Isabelle DesguerreReference Centre for Neuromuscular Diseases Nord/Ile de France/Est, Paediatric Neurology Department, Hôpital Necker-Enfants Malades, AP-HP, Paris, France.
Pascal CintasNeurology Department, Toulouse Purpan University Hospital, Reference Centre for Neuromuscular Diseases, 31059 Toulouse, France.ORCID 0000-0002-0242-4849
Carole VuillerotPediatric Physical Medicine and Rehabilitation Department, Hôpital Mère Enfant, CHU-Lyon, Lyon, France.
Frederic AudicPaediatric Neurology Department, Reference Centre for Neuromuscular Disorders and ALS, CHU La Timone, Marseille, France.
Claude CancesPediatric Neurology Department, Reference Centre for Neuromuscular Disorders, Toulouse Children's Hospital, Toulouse, France.ORCID 0000-0001-7386-2275
Jon Andoni UrtizbereaAcademyo Program Unit, Institute of Myology, Paris, France.
Shahram AttarianPaediatric Neurology Department, Reference Centre for Neuromuscular Disorders and ALS, CHU La Timone, Marseille, France.
Juliette RoparsClinical Research Unit, Assistance Publique - Hôpitaux de Paris University Paris-Saclay, Faculty of Medicine, University of Versailles Saint-Quentin en Yvelines, Inserm U1018 Team Anti-infective evasion and pharmacoepidemiology, Boulogne-Billancourt, France.ORCID 0000-0001-7467-759X
Susana Quijano-RoyPediatric Neurology and ICU Department, Garches Reference Centre for Neuromuscular Diseases (NEIF for FILNEMUS; RPC for Euro-NMD ERN), AP-HP Paris Saclay University, Hôpital Raymond Poincaré (UVSQ), 104 boulevard Raymond Poincaré, 92380 Garches, France.ORCID 0000-0002-8503-6111
Registre SMA France Study Group
Funding
No grant is acknowledged in the PubMed record.
6 · The paper itself
Abstract
BackgroundSpinal muscular atrophy (SMA) is a severe neurodegenerative disease affecting children. Three innovative disease-modifying therapies (DMTs)-nusinersen, risdiplam, and onasemnogene abeparvovec-are available for treatment.ObjectiveTo provide a descriptive overview of patients enrolled in the
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.
REGISTRE SMA FRANCE: A nationwide observational registry of patients with spinal muscular atrophy in France. · full record | OpenQuestion