We propose a selection of websites that will provide you with additional information on neuromuscular disorders.
The French Muscular Dystrophy Association (AFM) is composed of patients and their families who are affected by a genetic, rare, progressive and severely disabling illness: neuromuscular diseases. In order to fight those diseases, AFM chose to initiate innovative actions and a strategy of general interest that benefits all rare diseases and all persons with disabilities.
- Biotherapies Institute
The Biotherapies Institute unites the knowledge and expertise of four laboratories, initiated and supported by the AFM-Telethon, world leaders of biotherapies for rare diseases. Objective: to accelerate the development of treatments against rare diseases.
Created and funded by AFM-Telethon, Genethon’s mission is to design gene therapy products for rare diseases, to ensure their pre-clinical and clinical development as well as their production in order to provide patients with access to these innovative treatments.
Created in 2005 through a collaboration between Inserm – National Institute of Health and Medical Research – and AFM-Telethon – French Association against Myopathies – I-Stem is the largest French laboratory for research and development dedicated to human pluripotent stem cells, of embryonic origin or obtained by reprogramming gene. I-Stem is part of the Biotherapy Institute for Rare Diseases, which includes so far the four centers of research and development funded directly by the AFM Telethon.
Orphanet is the reference portal for information on rare diseases and orphan drugs, for all audiences. Orphanet’s aim is to help improve the diagnosis, care and treatment of patients with rare diseases.
- Institut Pasteur
The Institut Pasteur is a private, non-profit foundation with recognized charitable status entrusted with four core missions of public interest – Research, Education, public health, and Valorisation of scientific research via technology transfer and industrial partnerships.
- European Neuromuscular Centre (ENMC)
The mission of ENMC is to encourage and facilitate communication and collaboration in the field of neuromuscular research with the aim of improving diagnosis and prognosis, finding effective treatments and optimizing standards of care to improve the quality of life for affected people and their families.
- Treat-NMD Neuromuscular network
TREAT-NMD is a network for the neuromuscular field that provides an infrastructure to ensure that the most promising new therapies reach patients as quickly as possible. Since its launch in January 2007 the network’s focus has been on the development of tools that industry, clinicians and scientists need to bring novel therapeutic approaches through preclinical development and into the clinic, and on establishing best-practice care for neuromuscular patients worldwide.
EURORDIS is a non-governmental patient-driven alliance of patient organisations and individuals active in the field of rare diseases in Europe and beyond. EURORDIS’ mission is to build a strong pan-European community of patient organisations and people living with rare diseases, to be their voice at the European level.
- Clinical trials
ClinicalTrials.gov is a registry and results database of publicly and privately supported clinical studies of human participants conducted around the world.