Open Trials

Clinical Trial of Investigational Medicinal Products (CTIMP) 

• FOR-DMD (Steroid regimen for DMD)
• SRP-4053 (DMD)
• PF-06252616 (DMD)
• Ataluren (PTC124) (Nonsense mutation Dystophinopathy)
• Sialic acid (GNE Myopathy (GNEM) and Hereditary Inclusion Body Myopathy (HIBM))
• Bumetanide (Hypokalaemic periodic paralysis)
• Valproate Sodium (McArdle Disease)
• neoGAA (Pompe disease)
• ISIS 396443 (SMA)
• MOONFISH - RO6885247 (Spinal Musculal Atrophy (SMA))

Cohorts

• IBM-Net (Inclusion Body Myositis)
• Mys-IBM (Sporadic Inclusion Body Myositis)
• IBM-Genetic (Sporadic Inclusion Body Myositis)
• HIBM-PMP (Hereditary Inclusion Body Myositis (HIBM))
• Identification of susceptibility genes (Primary inflammatory muscle diseases, PM, DM and IBM)
• Investigation of ion channel disorder
• Global FKRP registry (Limb Girdle Muscular Dystophy type 2I and Congenital Muscular Dystrophy type 1)
• Bridge Neuropathic pain (NIHR Pain Consortium)
• GNE-DMP (GNE Myopathy)
• NorthStar (DMD)
• AFM Study outcome measures in DMD
• Becker Muscular Dystrophy - Natural history to predict efficacy of exon skipping
• UK Myotonic Dystrophy patient registry
• FSHD - NH study
• FSHD registry
• SMA REACH UK
• SMA registry
• Kennedy's Disease Study and Register
• Genotype-Phenotype in inherited neurodegenerative diseases
• MD-Core (Congenital muscular Dystrophies and congenital myopathies)
• IGOS (Guillain-Barre syndrome (GBS))

Other studies

• Exercise, cognition and brain vitality
• Aerobic training (IBM)
• Physical activity (IBM)
• Qualitative Magnetic Resonance Imaging (Channelopathies)
• Quantitative MRI to monitor disease activity (Hypokalaemic Periodic Paralysis)
• Exercise and Sarcopenia