FOR DMD is an NIH funded study to compare three ways of giving corticosteroids to boys with Duchenne muscular dystrophy to determine which increases muscle strength the most, and which causes the fewest side effects.
We coordinate a natural history and clinical outcomes study of dysferlinopathy (limb-girdle muscular dystrophy type 2B) which is funded by the Jain Foundation. This is an opportunity for patients with dysferlinopathies to participate in a clinical research study which is being performed in 14 centre’s in around the world including: France, Germany, Italy and the UK in Europe along with four sites in the United States, one each in Japan and Australia.
TROPHOS is a phase II, mulitcentre, randomised, adaptive, double blind, placebo controlled Safety and Efficacy of olesoxime (TRO19622) in 3-25 year old non ambulant Spinal Muscular Atrophy (SMA) patients.
Prosensa DMD Extension Study – DMD114349 is an open-label extension study of the long-term safety, tolerability and efficacy of GSK2402968 in subjects with Duchenne Muscular Dystrophy.
DMD Heart Protection Study: A double-blind randomised multi-centre, placebo-controlled trial of combined ACE-inhibitor and beta-blocker therapy in preventing the development of cardiomyopathy in genetically characterised males with DMD without echo-detectable left ventricular dysfunction.
A natural history study in Duchenne Muscular Dystrophy focussing on outcome measures funded by the AFM.
An Open-Label study for previously treated Ataluren (PTC 124®) Patients with Nonsense mutations Dystrophinopathy.
Prosensa 045: A phase I/IIa, open-label, escalating dose study to assess the safety and tolerability, pharmacokinetics, pharmacodynamics and clinical effects of multiple subcutaneous doses of PRO045 in subjects with Duchenne muscular dystrophy
“Hereditary Inclusion Body Myopathy-Patient Monitoring Program (HIBM-PMP): A Registry and Prospective Observational Natural History Study to Assess HIBM Disease” *** Reference to Ultragenyx***
A phase III efficacy and safety study of Ataluren (PTC124) in patients with nonsense mutation dystrophinopathy.
An open-label, multicenter, multinational, ascending dose study of the safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of repeated biweekly infusions of neoGAA in naïve and alglucosidase alfa treated late-onset Pompe disease patients.
A Phase I/II, open-label, dose escalating with 48-week treatment study to assess the safety and tolerability, pharmacokinetics, pharmacodynamics and efficacy of PRO053 in subjects with Duchenne muscular dystrophy