Trials ongoing at the Institute of Myology, divided into interventional research and non-interventional research.
Interventional research
Trials in interventional research concern biomedical research (BMR) with and without healthcare products. BMR covers all research organised and performed on humans for the development of biological or medical knowledge, during which drugs, medical devices, cell and gene therapy products, etc. are evaluated. The Institut is implied in:
- Spinal Muscular Atrophy: NatHis-SMA, AveXis, IONIS-CS11-SHINE, SUNFISH, FIREFISH
- Myotonic Dystrophies: Nathis-DM1, PsyDM1, Steinert
- Duchenne Muscular Dystrophy: WAVE, WAVE-OLE, Nathis-DMD, Pre-U7, SRP4053, Essence, SIDEROS, PTC-PASS, Italfarmaco, Anti-Myostatin, SRP 4658-102
- Limb-girdle muscular dystrophy: Dysferlinopathy
- Glycogen storage diseases: Pro-GSDIII, NeoGAA-TDR 12857, EO-EXT-LTS 13769,
- Becker Muscular Dystrophy: Becker-Coeur
- Hereditary inclusion body myopathy: HIBM-PMP, HIBM-Phase III
- Mitochondrial diseases: Mitochondrial diseases cohort follow-up
- Myotubular Myopathy: Nathis-CNM, INCEPTUS, ASPIRO
- Other disease: GLUT-HEP
Non-interventional research
This type of research is performed without any type of intervention; products used and all acts carried out are as normal, without any additional or unusual diagnostic or monitoring procedure. It may involve biological collection (collection of biological samples or by-products on selected subjects), data (use of data already collected) or it may be observational (no additional diagnostic or monitoring procedure). The Institut is implied in:
- Collagen VI myopathies: UMD-COL6 A1, UMD-COL6 A2, UMD-COL6 A3
- Duchenne Muscular Dystrophy: UMD-DMD
- Dynaminopathies: UMD-DNM2
- Laminopathies and Emerinopathies: UMD FHL1A, UMD FHL1B, UMD FHL1C, UMD-EMD, UMD-LMNA, OPALE
- Glycogen storage diseases: Myozyme, GDSIII
- Spinal Muscular Atrophy: Registry SMA
