Closed Trials

Clinical Trial of Investigational Medicinal Products (CTIMP)

• GSK Extension study (DMD)
• Restoring dystrophin (DMD)
• AVI-4658 (DMD)
• PRO051 - GSK2402968 (DMD)
• SMT C1100 (DMD)
• PTC124 (Duchenne and Becker muscular dystrophy)
• Arimoclomol (IBM)
• Mexiletine (Non-dystrophic myotonia)
• HYP HOP study (Dichlorphenamide for Periodic Paralysis)
• Olesoxime (TRO19622) (SMA)

Cohorts

• Complication of pregnancy (Mitochondrial disease)
• Jain Foundation limb-girdle muscular dystrophy type 2B
• Outcome measure in SMA type II and III
• OPTIMISTIC (Myotonic Dystrophy)
• Genotype - phenotype correlation and longitudinal studies (Non-dystrophic Myotonias)
• Genotype - phenotype correlation and longitudinal studies (Andersen-Tawil syndrome)
• Genotype - phenotype correlation and longitudinal studies (Episodic Ataxia Syndrome)

Other studies

• Causes of fall and balance impairment in people with neuromuscular diseases
• Exercise training in patients with Mitochondrial disease
• Cardiac adaptation to exercise in Mitochondrial disease
• Muscle imaging biomarkers (DMD)
• MRI in FKRP-Related LGMD2I
• MRI and MRS in FKRP-Related LGMD2I
• MRI in IBM and CMT