Project description
Nutritional management of myotonic dystrophy type 1
Myotonic dystrophy type 1 (DM1) is a multisystemic neuromuscular rare disease without cure or treatment available. A dietary management approach could provide a unique option for rare disease patients through foods for special medical purposes (FSMP). MYODM is a unique formulation of caffeine and theobromine especially developed to nutritionally manage DM1. However, as a food supplement, MYODM faces the problem that the DM1 community requires in human data for substantiating the use of the food product. Through a pilot interventional clinical trial, the EU-funded MYODM-FSMP project will compile in human evidence that MYODM is a unique approach to improving the quality of life for patients with DM1.
Objective
Myotonic Dystrophy type 1 (DM1) is the most common and multisystemic neuromuscular rare disease affecting 1 million people worldwide, 100,000 EU citizens, who still have no cure or treatment available.
In the light of the ´Leave No-one Behind´ commitment behind Sustainable Development Goals and particularly, the dietary management approach may provide a unique option for rare disease patients through Foods for special medical purposes (FSMP), foods (not drugs) intended for the dietary management of individuals who suffer from certain diseases. The main goal of the present project is to compile in human evidences that MYODM’s food for special medical purposes is a unique approach to improve DM1 patients’ Quality of Life through a pilot clinical trial for foods and supplements sponsored by Myogem and leaded by Dr. Adolfo López de Munain, a worldwide neurologist specialized in DM1.
MYOGEM is the current licensee of the patents WO2016075288 A1 and WO2016075285 A1, which disclosed the effects of some methylxanthines on DM1. Owning the Intellectual Property Rights over the present solution is one of the main competitive advantages of MYOGEM. MYOGEM is the first and unique company worldwide who launched a food supplement for a rare disease in May 2017. However, as food supplement MYODM faces the problem that DM1 community, either neurologists and patient associations, claim for in human data in order to broadly prescribe and consume the product. To overcome this barrier, and gain market confidence, MYOGEM aims to perform a Feasibility pilot clinical trial to demonstrate MYODM’s effect on a small cohort of 10-15 DM1 patients in order to commercially upgrade our current product “food supplement” to “food for special medical purpose”, from now on referred as MYODM-FSMP, that will reach the market in less than one year after the present project ends.
By launching the new MYODM-FSMP product, a 5.5 M€ SOM is expected in 5 years after the project. Two SoE have been awarded.
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Programme(s)
Funding Scheme
SME-1 - SME instrument phase 1Coordinator
08100 Mollet Del Valles
Spain
The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.