Grant Funding

Joint Research Call with Great Ormond Street Hospital Charity (GOSH) 2026/27

Great Ormond Street Hospital Charity has announced their 2026/27national funding call in child health research. Under this funding call £2.5M is available to support eligible child health research project grant applications from researchers across the UK. The project budget for individual proposals is £350,000 and we are delighted that this year, Myotubular Trust is again included in the four rare disease charities partnering with the GOSH charity.

Remit of the National Research Project Grants

GOSH Charity is inviting project grant applications for paediatric health research studies focusing on complex or rare diseases. The call will support research across the spectrum of medical conditions affecting the foetus, neonates, and children directly, as well as pregnancy disorders that affect the child.

Within this call they are specifically asking for applications that aim to significantly advance the search to find a cure and/or a treatment for any of the genetic forms of centronuclear and myotubular myopathy, and studies that would increase an understanding of liver abnormalities in the condition.

All projects must aim to improve understanding of the disease or associated conditions or to improve outcomes for the affected child. Proposals will be expected to have the potential to lead to new medical developments or improved healthcare (e.g. diagnostic tools or novel interventions) through laboratory and/or clinically based research. Qualitative and mixed-methods research projects, including healthcare management for specific childhood diseases/conditions or groups of closely related conditions, are also welcomed. Applicants must clearly articulate the route to clinical application and/or potential for patient benefit, regardless of where their proposed studies are positioned along the basic-to-applied research continuum.

The deadline for submission of outline applications is Thursday, 16th July 2026 at 3 PM.

We are proud to be part of a partnership with this world renowned charity which has done so much to further the health of children, and we thank all our donors and fundraisers who make it possible for us to be part of such a partnership. See here for more information.

Our Funded Projects

An advanced multi-organoid platform for therapy modelling in x-linked myotubular myopathy – Professor Francesco Saverio Tedesco – £248,940 over 3 years (This grant is awarded in partnership with Great Ormond Street Hospital Charity)

Understanding liver disease in XLMTM – Dr James Dowling, Hospital for Sick Children, Toronto, Canada – £300,000 over 3 years (This grant is awarded in partnership with Muscular Dystrophy UK)

Next generation AAV-mediated gene therapy for myotubular myopathy and pathophysiology – Dr Ana Buj Bello, Inserm, France – £135,377 over 2 years

Is the mTORC1 pathway a route to treatment in x-linked myotubular myopathy? – Dr Karim Hnia, Inserm, Toulouse, France – £82,000 over 2 years

TAM4MTM-UK: Safety and Efficacy of Tamoxifen Therapy for Myotubular Myopathy in the UK – Dr Giovanni Baranello, UCL Great Ormond Street Institute of Child Health, London – £232,433 over 3 years (This project is co-funded with Great Ormond St. Hospital Children’s Charity (GOSHCC) and Sparks, the children’s medical research charity)

TAM4MTM-UK – preparing the regulatory and ethical paperwork and processes for the clinical trial – A part time clinical trial co-ordinator at UCL Great Ormond Street Institute of Child Health, London – £6,104 over 6 months

Combinatorial therapy as a novel treatment strategy for myotubular myopathy (MTM1) – Dr James Dowling, Hospital for Sick Children, Toronto, Canada – £216,000 over 3 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

PI3 Kinase inhibition as a novel treatment strategy for myotubular myopathy (MTM1) –Dr James Dowling, Hospital for Sick Children, Toronto, Canada – £162,500 over 3 individual one year grants

Reducing DNM2 as a novel therapeutic target for centronuclear myopathy– Dr Jocelyn Laporte, IGBMC, France – £132,000 over 3 years

Pre-clinical gene replacement therapy for X-linked myotubular myopathy (MTM1) – Dr Ana Buj Bello, Genethon, Evry, France – £110,300 over 2 years

Secondary pathogenic mechanisms in XLMTM and CNM – Dr Susan Treves, Basel University Hospital, Basel, Switzerland and Professor Heinz Jungbluth, King’s College London – £116,664 over 3 years

Next generation sequencing to tackle centronuclear myopathies – Dr Jocelyn Laporte, IGBMC, France – £62,000 over 2 years

Gene therapy for autosomal dominant centronuclear myopathy by Transplicing – Dr Marc Bitoun, INSERM, Paris – £102,340 over 2 years

Deciphering the molecular pathway involving centronuclear myopathy genes – Manuela D’Alessandro, IGBMC, Illkirch, France – £120,000 over 3 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

Gene therapy for x-linked myotubular myopathy and pathophysiology – Dr Ana Buj Bello, Genethon, Evry, France – £102,290 over 2 years

The Research Process

Please feel free to download the pdf explaining more about the Myotubular Trust’s research selection process.