Why use the BioBank?
The basic scientific research being carried out on neuromuscular diseases relies on the availability of high-quality biomaterials (DNA, cells and tissue), while clinical trials rely on the availability of suitable patient cohorts. For new treatments to make their way into clinical practice for patients affected with neuromuscular disorders, it is essential that access to biomaterials is facilitated. The improvement of supranational biobanks and the introduction of European patient databases for DMD, SMA and other neuromuscular diseases are therefore major goals of this integrating activity within TREAT-NMD.
Developing and managing international biobanks
Before new, innovative therapeutic strategies can be applied to patients, scientists have to perform numerous preclinical experiments, including tests on biomaterials, such as muscle cells. The idea of a supranational biobank is to provide a network of biobanking facilities that will encourage the storage of biomaterials for NMD patients and help scientists to obtain more easily the specific material they need for their experiments on neuromuscular diseases. Therefore, one goal of TREAT-NMD is to improve the availability and the exchange of biomaterial among scientists across Europe, in collaboration with the already existing EuroBioBank network.
Examples of how the BioBank is being used
- The clinical outcome study for dysferlinopathy is an international clinical research study that aims to define outcome measures for clinical trials. Read more
- The MYO-SEQ project is collaborative study applying whole exome sequencing to a large cohort of patients with unexplained limb‐girdle weakness
- PhenoDM1 is a deep phenotyping study of Myotonic Dystrophy type 1 (DM1), aiming to improve delivery of personalized medicine and assist in the planning, design and recruitment of clinical trials
- As part of an ancillary study running alongside the FOR-DMD trial all participating boys will be asked if they are willing to provide blood samples that would be stored in the EuroBioBank. Read more
- BIO-NMD is a research project which has been looking for biomarkers in patients with neuromuscular disease. Read more