Research

For clinicians and researchers, lack of support tools such as validated clinical outcome measures or standard operating procedures for research protocols has held back therapeutic development. Whilst for patients, promising research results
have still not been translated into the treatments they hope for.

Strong collaborations have been built across the neuromuscular community by engaging numerous international researchers and organizations worldwide, who are able to tap into the resources developed by TREAT-NMD. The resources provided in this section of our site span the research and clinical arenas and are part of a whole suite of tools that are essential for rapid progress towards new therapies.

Protocols for research on animal models

Download standardized operating procedures (SOPs) for experimental protocols for animal models of DMD and SMA, drawn up by specialists from all over the world and designed to improve comparability of studies

BioBanking

This unique network of twelve biobanks from seven EU countries stores and distributes quality DNA, cell and tissue samples for scientists conducting research on rare diseases, including neuromuscular
disorders

 

Clinical research

Clinical research includes both clinical trials that test new treatments and natural history studies, which provide valuable information about how diseases progress

Outcome measures

"Outcome measures" are the tests that investigators perform to decide whether a treatment being tested in a clinical trial is having any effect