Descrizione del progetto
Gestione nutrizionale della distrofia miotonica di tipo 1
La distrofia miotonica di tipo 1 (DM1, myotonic dystrophy type 1) è una malattia rara neuromuscolare multisistemica senza cure o trattamenti disponibili. Un regime dietetico potrebbe costituire un’opzione unica per i pazienti affetti da malattie rare attraverso alimenti a fini medici speciali (FSMP, foods for special medical purposes). MYODM è una formulazione unica di caffeina e teobromina sviluppata appositamente per la gestione nutrizionale della DM1. Tuttavia, in quanto integratore alimentare, l’uso del prodotto alimentare MYODM deve essere comprovato dalla comunità DM1, che richiede dati sull’uomo a tale scopo. Attraverso uno studio clinico interventistico pilota, il progetto MYODM-FSMP, finanziato dall’UE, raccoglierà testimonianze umane relativamente all’unicità dell’approccio MYODM per migliorare la qualità della vita dei pazienti affetti da DM1.
Obiettivo
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.
Campo scientifico
Parole chiave
Programma(i)
Argomento(i)
Invito a presentare proposte
Vedi altri progetti per questo bandoBando secondario
H2020-SMEInst-2018-2020-1
Meccanismo di finanziamento
SME-1 - SME instrument phase 1Coordinatore
08100 Mollet Del Valles
Spagna
L’organizzazione si è definita una PMI (piccola e media impresa) al momento della firma dell’accordo di sovvenzione.