Facilitating research networking
TREAT-NMD is bringing scientists together in many different areas – from facilitating exchange on high throughput screening methodologies to optimizing production and safety of therapeutics. In one example, researchers working on different antisense oligonucleotide chemistries in different labs (the MDEX consortium led by Professor Francesco Muntoni in London and the Leiden lab led by Dr Annemieke Aartsma-Rus) have been able to set up a regular dialogue to discuss progress and results in a constructive and non-competitive way. This model for collaboration helps groups learn from one another and progress more quickly, without damaging their competitive advantage.
In other examples, the TREAT-NMD-integrated resource EuroBioBank is the only biobank network dedicated to rare disease research in Europe, and its samples are distributed to researchers across the globe.
TACT, the TREAT-NMD Advisory Committee for Therapeutics, is an expert multidisciplinary body that provides the neuromuscular community (clinicians, researchers, patient advocacy groups and industry) with independent and objective guidance on advancing new therapies (whether novel or repurposed) for neuromuscular diseases.
Comparability of results between different research groups is a major issue in the preclinical field. As the result of international collaboration between animal model specialists worldwide, consensus publications for DMD and SMA animal models and standard operating procedures for their evaluation have been published, and a similar process is currently ongoing for models for the congenital muscular dystrophies.
Consensus development is also ongoing in the area of outcome measures research, with the aim of selecting the most appropriate outcome measures for each condition at each stage of progression of the disease.