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The Myotubular Trust’s Research Strategy is to fund proof of principle translational research projects that will significantly advance the search to find a cure and/or a treatment for any of the genetic forms of centronuclear and myotubular myopathy.
We are delighted to announce that Myotubular Trust and the renowned children’s medical research charity, Action Medical Research, are teaming up to fund a project grant in 2018 for translational research into myotubular and centronuclear myopathy. Applications must meet the remit of both charities and all outline applications will be judged in open competition on both potential clinical impact, and scientific quality.
Applications may be made for project grants of up to 3 years in duration. Up to £200,000 of joint funding is available to spend on these co-funded projects in 2018. Principal investigators and applicants must be based at UK institutions and awards can only be made to UK institutions. Researchers outside the UK can collaborate on UK based projects if appropriate.
The application process will be managed through the Action Medical Research 2018 grant round and the outline application form can be found here. Successful applicants from the outline stage will then be invited to complete a full application for full external peer review in open competition with other applications in the Action Medical Research grant round.
The Myotubular Trust (Registered Charity No: 1137177) was set up in 2006 to raise money for research to find a cure and / or treatment for myotubular myopathy.
There are three genetically distinct forms of myotubular myopathy. The commonest is x-linked, affecting only boys, and also the most severe. It usually presents in the newborn period and there are associated breathing and swallowing difficulties in addition to the general muscle weakness. The other forms are either dominant or recessive in inheritance, are usually milder and vary widely.
Gene therapy for x-linked myotubular myopathy and pathophysiology – Dr Ana Buj Bello, Genethon, Evry, France – £102,290 over 2 years
Membrane trafficking and T tubule structure and function in a canine model of centronuclear myopathies– Dr Richard Piercy, Royal Veterinary College, London – £38,548 over 3 years
Deciphering the molecular pathway involving centronuclear myopathy genes – Manuela D’Alessandro, IGBMC, Illkirch, France – £120,000 over 3 years
Gene therapy for autosomal dominant centronuclear myopathy by Transplicing – Dr Marc Bitoun, INSERM, Paris – £102,340 over 2 years
Next generation sequencing to tackle centronuclear myopathies – Dr Jocelyn Laporte, IGBMC, France – £62,000 over 2 years
Secondary pathogenic mechanisms in XLMTM and CNM – Dr Susan Treves, Basel University Hospital, Basel, Switzerland and Dr Heinz Jungbluth, King’s College London – £116,664 over 3 years
Pre-clinical gene replacement therapy for X-linked myotubular myopathy (MTM1) – Dr Anna Buj Bello, Genethon, Evry, France – £110,300 over 2 years
Reducing DNM2 as a novel therapeutic target for centronuclear myopathy– Dr Jocelyn Laporte, IGBMC, France – £132,000 over 3 years
PI3 Kinase inhibition as a novel treatment strategy for myotubular myopathy (MTM1) –Dr James Dowling, Hospital for Sick Children, Toronto, Canada – £102,500 over 2 years
Myotubular and Centronuclear Myopathy Patient Registry curation –Professor Hanns Lochmueller, TREAT-NMD, John Walton Muscular Dystrophy Research Centre, Newcastle University – £60,744 over 2 years
Combinatorial therapy as a novel treatment strategy for myotubular myopathy (MTM1) – Dr James Dowling, Hospital for Sick Children, Toronto, Canada – £216,000 over 2 years
Please download an application forms in either PDF or Word Document here:
Myotubular Trust Project Grant Application (word document)
Myotubular Trust Research Fellowship Application (word document)
Should you have any questions relating to this call for projects, please email email@example.com.
Please feel free to download the pdf explaining more about the Myotubular Trust’s research selection process.