Research tools

In order to develop innovative treatments you must have the right tools: vector production centres, screening platforms, databases as well as DNA and tissue banks.

Illustration for article: Screening platforms

Screening platforms

AFM-Téléthon is striving to discover effective treatments. This involves research to develop new drugs. Screening platforms are a valuable tool to accelerate the discovery of these candidate-drugs.
Illustration for article: Vectors and meganucleases: explanations

Vectors and meganucleases: explanations

Vectors are gene-drug "shuttles" whereas meganucleases are "DNA scissors". They are tools which are essential for gene therapy and gene surgery. This is why the French Muscular Dystrophy Associationg (AFM-Téléthon) supports their production.
Illustration for article: Production of drug vectors

Production of drug vectors

The emergence and first results of biotherapies demonstrates the efficacy of AFM-Téléthon’s strategy. The next bold challenge which has never been taken up by a patients’ association is the production of biodrugs and clinical development.
Illustration for article: Cell production and storage

Cell production and storage

Cells are a basic tool for research and cell therapy. They must therefore be produced and studied from every angle to understand why they are defective or if they display good therapeutic potential.
Illustration for article: Reference databases

Reference databases

With the computerisation of medical data, databases are growing fast. They provide a terrific resource for research because they lead to a significant improvement of knowledge about diseases. The French Muscular Dystrophy Association (AFM-Téléthon) helps producing genetic and clinical databases to improve patient recruitment for clinical trials for instance.
Illustration for article: DNA and tissue banks: keys to research

DNA and tissue banks: keys to research

Banks of samples of biological material (tissues, cells, DNA, etc.), called biobanks, provide researchers with essential human biological material for their research. Some of them are dedicated to rare diseases, particularly neuromuscular diseases. They include biological resources obtained from voluntary and anonymous donations made by people suffering from these diseases. This is the case of the Généthon DNA and cell bank and the Myobank-AFM/Institut de Myologie tissue bank, both created by the French Muscular Dystrophy Association (AFM-Téléthon) and authorised and recognised by the French Ministries of Research and Health.
Illustration for article: Experimentation and exploration platforms

Experimentation and exploration platforms

Research requires good tools for exploration and experimentation. AFM-Téléthon supports teams which develop tools for evaluating muscle function or helps conduct preclinical trials required by regulatory agencies in animal models before moving on to human trials.