Descripción del proyecto
Gestión nutricional de la distrofia miotónica tipo 1
La distrofia miotónica tipo 1 (DM1) es una enfermedad neuromuscular multisistémica rara sin cura ni tratamiento disponible. Un método de gestión nutricional podría ofrecer la única opción para los enfermos con esta enfermedad rara a través de alimentos para usos médicos especiales (FSMP, por sus siglas en inglés). MYODM es una formulación única de cafeína y teobromina desarrollada especialmente para la gestión nutricional de la DM1. Sin embargo, como complemento alimenticio, MYODM afronta el problema de que la comunidad de la MD1 necesita datos de estudios en humanos para justificar el uso de este producto alimenticio. A través de un ensayo clínico de intervención piloto, el proyecto financiado con fondos europeos MYODM-FSMP recopilará pruebas en humanos de que MYODM es un método único para mejorar la calidad de vida de los pacientes con MD1.
Objetivo
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.
Ámbito científico
Palabras clave
Programa(s)
Convocatoria de propuestas
Consulte otros proyectos de esta convocatoriaConvocatoria de subcontratación
H2020-SMEInst-2018-2020-1
Régimen de financiación
SME-1 - SME instrument phase 1Coordinador
08100 Mollet Del Valles
España
Organización definida por ella misma como pequeña y mediana empresa (pyme) en el momento de la firma del acuerdo de subvención.