Projects

PATIENT REGISTRY

 

The National Patient Registry is the database gathering the main characteristics of patients affected with CMD1A all over Spain.

 

Currently, in Spain, there is no other registry of people affected with CMD1A. Creating this registry has many benefits, because it helps to discover how many people we are, and which are our main features. The importance of this information lies on its usefulness, on one side for developing clinical studies with patients, and on the other side to characterise the disease.

The registry we are developing is compatible with the one of Cure CMD, the organisation gathering the majority of patients with congenital muscular diseases.

 

For more information about our patient registry, don’t hesitate to contact us in  registro@impulsate.org

We’ll be delighted to hear from you.

DEVELOPMENT OF A NOVEL PRECLINICAL GENE THERAPY APPROACH FOR CONGENITAL MUSCULAR DYSTROPHY 1A

 

ImpulsaT collaborates on the funding of the project “Development of a novel preclinical gene therapy approach for congenital muscular dystrophy 1ª”, led by Dr. Jordi Barquinero, head of the Gene and Cell Therapy at VHIR, and Dr. Francina Munell, research doctor specialized in paediatric neurology and neuromuscular diseases in VHIR. This research focuses on the development of genetic therapies which enable the expression of laminina alfa-2 (merosin), thus, allowing the protein to develop its function. The project is co-financed by ImpulsaT and the City Council of Castellbisbal.

UNI-LARGE: ADVANCED GENE EDITING TECHNOLOGIES

 

We also contribute to the project “Uni-large: Advanced Gene Editing Technologies”, led by Dr. Marc Güell, head of the synthetic biology group at PRBB (UPF), in the frame of the program “CaixaImpulse”. The research of the group is focused on Uni-large, a new strategy of gene editing technology which combine CRISPR/cas9 precision and the efficacy of viral vectors established in clinical trials. They are developing a first therapy to treat congenital muscular dystrophy type 1A (DMC1A). As laminina alfa-2 protein exceeds the size limit of other vectors, it is necessary to develop alternative therapies. For this reason, new steps are being taken for the preclinical development of this technology, in order to ensure its safeness when treating human muscular disorders and other diseases.