| QUIJANO-ROY, Susana |
NusiMFM, NCT04602195: Responsiveness and Validation Study of MFM-32 in SMA Patients Treated With Nusinersen |
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| Completed | N/A | 33 | Europe | | Hospices Civils de Lyon | Spinal Muscular Atrophy | 11/23 | 11/23 | | |
OPALE, NCT03058185: Observatoire Des Patients Atteints de Laminopathies et Emerinopathies (Observatory for PAtients With Laminopathies and Emerinopathies) |
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| Recruiting | N/A | 800 | Europe | | Pitié-Salpêtrière Hospital, Institute of Myology, Assistance Publique - Hôpitaux de Paris, Institut National de la Santé Et de la Recherche Médicale, France | Laminopathies, Emerinopathies | 07/33 | 07/33 | | |
PHENO SMART, NCT06321965: Characterization of New Phenotypes of Patients with Spinal Muscular Atrophy Treated with SMN Restoring Therapy |
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| Recruiting | N/A | 60 | Europe | evaluation of muscle function, First-line cognitive assessment, second-line cognitive assessment, Cardiac evaluation, MRI, Assessment of activity and muscle fatigue, Assessment of bulbar function, Evaluation of body composition and metabolism, Questionnaires, Biocollection, Skinfold measurement | Hospices Civils de Lyon | Spinal Muscular Atrophy | 01/28 | 01/28 | | |
| Not yet recruiting | N/A | 15 | Europe | Mobility study in dry conditions, Mobility study in water conditions, Garches Bath Test (GBT) | Assistance Publique - Hôpitaux de Paris | Infantile Spinal Muscular Atrophy | 11/25 | 11/25 | | |
| Recruiting | N/A | 40 | Europe | Skin Biopsy, Muscle biopsy | Institut National de la Santé Et de la Recherche Médicale, France | Laminopathies, Emery Dreifuss Muscular Dystrophy 2, LMNA-Related Congenital Muscular Dystrophy, Dilated Cardiomyopathy-1A | 09/27 | 12/27 | | |
| Recruiting | N/A | 1000 | Europe | | Assistance Publique - Hôpitaux de Paris | Spinal Muscular Atrophy | 01/29 | 01/29 | | |
| Not yet recruiting | N/A | 40 | Europe | | Hospices Civils de Lyon | Spinal Muscular Atrophy | 03/21 | 03/21 | | |
