The French Muscular Dystrophy Association (AFM) federates patients with neuromuscular diseases and their parents. Thanks in great part to donations from France’s annual Telethon, the AFM-Telethon has become a major player in biomedical research for rare diseases in France and worldwide. It currently funds about 37 clinical trials in different genetic diseases affecting the eye, blood, brain, immune system, and muscles… Thanks to its Genethon research lab, the AFM-Telethon stands out through its unique ability to produce and test its own gene-based medicines.
SMACARE Association is a non-governmental and non-profit organisation that aims to protect the rights and interests of people affected by spinal muscular atrophy and to improve their lives. The association was founded at the initiative of some parents whose daughter is affected by Spinal Muscular Atrophy Type II. Helped by relatives and friends and encouraged by doctors decided to start on this road to change the mentality of Romanians about people with disabilities, to make this disease known and to build a communication network between people with muscular spinal atrophy, their families and doctors.
Asociatia SMACARE aims to:
CSMA unites both parents of children and people with spinal muscular atrophy (SMA) to foster exchanges of ideas in order to solve, at least partially, existing problems. CSMA also supports the maintenance of a national registry of patients with SMA and provides information for care.
The German Society for people with Muscular Diseases (Deutsche Gesellschaft für Muskelkranke) was founded in 1965 as a result of parents’ initiative. Its main objective is the research into and treatment of muscle disorders. DGM includes more than 8,000 members, making it not only the oldest but also the largest patient organisation for people with neuromuscular diseases in Germany. DGM provides advice, assistance and the opportunity to exchange experiences with other stakeholders. Moreover, DGM is committed to the concerns of the stakeholders in the area of health policy and specifically promotes research in the field of neuromuscular diseases.
The Association of Families of SMA is a non-profit NGO founded in 2001 by a group of parents of children with SMA. The Association is a point of reference for medical and scientific researchers and for all the families of children with SMA. It aims to inform families about progress in scientific research, available treatments and assistance to which they are entitled to from public institutions; to promote and support scientific research in SMA and possible therapies; to communicate developments in clinical trials of drugs and therapies as well as the participation of Italian research organisations in clinical trials for SMA abroad, also solving legal problems, bureaucratic and organisational related to such participation.
FSMA Iceland was formally established in 2002 and is an association of families and individuals who suffer from Spinal Muscular Atrophy (SMA). Its purpose is to protect the interests of the persons with SMA and their families and to contribute to finding a cure. FSMA does this by fundraising to support research into the science of the disease; providing information to members of FSMA in Iceland on the progress of research on the disease, as well as other useful information related to the disease; to hold meeting on issues SMA; to disseminate information in the media and to the media about issues pertaining to SMA; to contribute to the transportation of individuals with SMA and their families.
The Polish foundation was formed in 2013 by parents of children with spinal muscular atrophy (SMA) to build a support network. The Foundation’s goals are to conduct activities for people suffering from SMA and their loved ones, aimed at combating exclusion, increasing independence and improving their quality of life; increasing awareness of SMA, in particular the dissemination of knowledge in genetics, diagnostics, standards of care and treatment methods; increasing the availability of methods and techniques for diagnostic, therapeutic, rehabilitative and related products and technology solutions; support system solutions, particularly in health care and social security, taking into account the needs of people suffering from SMA and their loved ones.
Spinal Muscular Atrophy Foundation (FUNDAME) is a non-profit, private foundation, established in 2005 and made up of patients affected by SMA and their relatives. FUNDAME strives to find ways to improve the quality of life of those affected by SMA and to promote research into this disease. FUNDAME supports research at both national and international level, in order to bridge the gap between today and the day a cure for SMA is available. In the meantime, FundAME seeks ways to improve the quality of life of those affected by the disease.
Nätverket för spinal muskelatrofi (NSMA) is a patient association which aim to bring together people who work and live with spinal muscular atrophy (SMA) in order to exchange, assist and inform; to be the natural platform for exchange and support for people living or working with SMA in Sweden; to raise awareness about SMA in Sweden; to influence public opinion and policy makers in different social and health care organisations on issues related to SMA; to establish a Swedish care program for the SMA with information and guidelines for different treatments.
The Prinses Beatrix Spierfonds is a foundation for over 200.000 people with a neuromuscular disease in the Netherlands. Our goal is to eliminate all neuromuscular diseases, by means of scientific research. We therefore finance and stimulate research aiming to develop therapies for neuromuscular diseases. Because this can be a long and strenuous process, we simultaneously support research in improving patient quality of life. In the past five years we invested over € 1 million in SMA research.
SMÁci, the Czech SMA Patient Organisation, was established in late 2016 and registered in March 2017, in response to growing demand for coordination of activities for SMA in the region.
The timing was opportune, as the landscape for SMA was changing so rapidly (approval of the first therapy, trials for other compounds, other activities etc.)
By 2018, SMÁci has 100 members and is still growing, albeit now at a slower rate, as the majority of patients/families have already joined.
Our organisation aims to support patients and their families with their quest for SMA. We strive to establish a communication channel between all parties involved and help to achieve a smooth execution of all available steps to improve quality of life.
The organisation succeeded in bringing Spinraza™ to patients in record-breaking time. Instead of the usual 3-7 years it takes in the Czech Republic, Spinraza™ reached patients within 1/2 a year from registration. Currently (summer 2018), 30% of all SMA patients are receiving the treatment.
Within the region, we are members of most relevant groups and organisations. In 2018 we also become an observer member of SMA Europe.
SMA Family Foundation Russia is a nonprofit organization that supports and empowers families with people affected by Spinal Muscular Atrophy. It was established in 2014 by parents of children with SMA for advocating for better services, raising public awareness and funding family’s special needs that are not reimbursed by national healthcare service. Our main aims are to build strong fundament for SMA Community to improve quality of life for individuals with SMA and their families,to fulfilling the strong need of information and best care practicies, to promote the development of medical and non-medical care for patients with SMA. Foundation also maintain the registry of SMA patiens in Russia.
SMA Schweiz is a non-profit swiss patient organisation which operates to develop and optimise therapies for people with spinal muscular atrophy (SMA). The goals of SMA Schweiz are to strengthen the patients’ voice in the drug development processes, to collaborate with various stakeholders to optimise the drug-development path from the laboratory to the patient, and to educate people affected by SMA and their families but also the public.
“SMA Serbia“ is a non-profit, non-governmental association, established in 2016 by parents of children affected by SMA and adults SMA patients. Our main goal is to increase the quality of life of those affected by SMA and their families. Our association strives to raise public awareness, protect the interest and the rights of patients, improve their social care and medical support. The idea is to build a community for Spinal Muscular Atrophy families and individuals, advise them and make their lives easier. Our final intention is to get the right treatment for every single patient, child and adult, so we can watch them grow, improve and prosper in many fields in life.
SMA UK was established in 1985 by a mother whose baby died aged 7 months from Spinal Muscular Atrophy Type 1. She set up the charity under the name of ‘Jennifer Trust for Spinal Muscular Atrophy’, now SMA UK, to offer support and hope to other families affected by the different types of SMA. Thirty years on, the charity continues to support and empower those affected by SMA. It advocates for better services and access to new treatments so that people affected by SMA are empowered and enabled to live full lives. It is committed to help fund and facilitate research and to raise public and professional awareness of SMA.
The Association of persons with Spinal Muscular Atrophy STOP SMA is a voluntary, non-profit, non-governmental association of citizens, established for the purpose of realising, protecting and promoting the rights and interests of the persons that suffer from SMA and their custodians, as well as their rights and interests in the area of health protection, the right to treatment, social protection, education and labor market.
VSN, The Dutch Neuromuscular Disease Association
The Vereniging Spierziekten Nederland (VSN), is an active and dynamic association of and for people with a neuromuscular disease. Its activities consist of providing information, organising mutual support and stimulating scientific research. The VSN favours international cooperation in the field of research and the development of therapies.
The VSN was founded in 1967 and now has approximately 9.000 members. More than 300 volunteers actively support the association’s activities.The VSN maintains a network of 10 regional groups and 15 national, diagnosis-bound support groups. These groups monitor international developments in the field of research, are involved in psychosocial care and provide information on technical appliances.
These groups inform their members on recent medical developments and initiate relevant research. They also publish brochures, leaflets and newsletters to inform the members.
An important part of the Association’s initiatives concerns the improvement of social and medical care for people with neuromuscular diseases. Relevant experts closely cooperate with the Association to improve diagnostic procedures, rehabilitation and genetic counselling. Concentration of care in specialised centres improves the level of care. Current areas of attention are registration of patient data, development of medical protocols/standards of care, and technical appliances.
Information: materials, webcasts, meetings, conferences
The VSN publishes many booklets, brochures and leaflets. They mostly concern diagnosis-specific information but also inform on general subjects such as education, respiratory support, or physiotherapy. Special information packages are available for medical professionals. Conferences, webcasts and meetings are organized yearly with more than 4.000 partcipants every year.