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13 April 2017 - POR FESR 2014-2020: Linea innovazione (Innovation line)

The Italfarmaco's project "ITF-Becker a new therapeutic approach at the Becker Muscular Dystrophy (BMD)"

The Italfarmaco's project "ITF-Becker a new therapeutic approach at the Becker Muscular Dystrophy (BMD)" which was developed in partnership with OPIS srl (Clinical Research Organization with 10 locations across Europe) and Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico with Neurology department, Neuromuscular and Rare disease department, and Neuroradiolgy unit, is among the 12 projects which have been selected in the Lombardia Region Call "Agreements on Research and Innovation" (DGR n. 5245 on 31st May 2016 and s.m.i) that will receive support by the Lombardia Region during the regional operative program 2014-2020 which the objective is "Investments in favor of growth and employment".

The Italfarmaco's project "ITF-Becker a new therapeutic approach at the Becker Muscular Dystrophy (BMD)" which was developed in partnership with OPIS srl (Clinical Research Organization with 10 locations across Europe) and Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico with Neurology department, Neuromuscular and Rare disease department, and Neuroradiolgy unit, is among the 12 projects which have been selected in the Lombardia Region Call "Agreements on Research and Innovation" (DGR n. 5245 on 31st May 2016 and s.m.i) that will receive support by the Lombardia Region during the regional operative program 2014-2020 which the objective is "Investments in favor of growth and employment".
The project is located in the regional specialization area S3 " Health Industry" with different objectives among those to improve the understanding of the natural history of the Becker Muscular Dystrophy (BMD) and to conduct a clinical study in BMD patients to evaluate the efficacy and safety of an investigational medicinal product "givinostat" in this type of dystrophinopathy, researching also some disease biomarkers. The clinical study will be managed in collaboration with OPIS, who will be accountable for the clinical and medical monitoring, for the management of the clinical data and to perform the statistical analysis. The Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico will be engaged to conduct the study, to recruit and treat the patients, to perform the medical evaluation, and to perform the histopathology and imaging analysis.

BMD is a rare neuromuscular disease, belonging to the group of dystrophinopathies, where Duchenne Muscular Dystrophy (DMD) is the most serious and disabling form. BMD is diagnosed between 10 to 30 years of age, only males are affected and it is characterized by progressive muscular weakness resulting in loss of ambulation generally between 20 to 45 years old and a significantly reduction in life expectancy due to a progressive dilated cardiomyopathy.
Some cases are milder with only muscular pain and sometimes with increase of blood CK values. There isn't currently BMD specific therapy: the therapeutic approach is multidisciplinary and primarily aimed at reducing or delaying possible complications.

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