The SMA Trust has spent time during the past year building closer relationships with other SMA organisations around the world. Two of our closest friends areThe SMA Foundation (based in New York), and The Jennifer Trust which is just down the road from us, in Stratford upon Avon. We are also actively involved with SMA Europe which is gaining some real momentum in co-ordinating efforts to attract scientists to research into SMA.
THE SMA FOUNDATION
The SMA Foundation is a US-based SMA charity whose mission is to accelerate the development of a treatment or cure for SMA, and which is dedicated to preventing the death and suffering of thousands of children whose lives depend upon increasing research funding to fulfil the scientific opportunity to cure the disease.
The mission of the SMA Foundation is to accelerate the development of a treatment.
Its fourfold strategy to achieve this includes:
- building entrepreneurial collaborations with academic and industry discovery efforts
- funding novel preclinical and research into SMA
- informing legislators, government agencies and the general public about the need for focus and support of SMA research
- bridging the gap between clinicians, scientists, technology and the pharmaceutical industry
Since 2003 the SMA Foundation has allocated more than $30 million to the support of basic, translation and clinical research that will accelerate progress towards a treatment in the near term, with special attention given to pilot studies and projects using novel approaches to SMA.
Research partners of the Foundation currently include Columbia University, The Massachusetts General Hospital, The Ohio State University, Stanford University, Inserm and the University of Wurzburg. Corporate partners include CombinatoRx, Inc., Curis, Inc.,Regeneron Pharmnaceuticals, Inc., PsychoGenics, Inc., and PTC Therapeutics, Inc.
THE JENNIFER TRUST FOR SMA
“Help for Today & Hope for Tomorrow”
The SMA Trust has had a close relationship with the JTSMA for a number of years and has provided financial support for its research projects.
The Jennifer Trust provides a range of support services for families of those affected by SMA. Its website is a rounded resource providing basic factual information to help SMA families, and also reports on the status of SMA research.
The JTMSA provides special equipment to sufferers and sustains a network of support families and groups to be available to people affected by SMA. An annual conference is held to bring together SMA families.
The SMA Trust is a member of SMA Europe the umbrella coalition of SMA patient organisations in Europe. SMA Europe was set up in 2006 in order to facilitate dialogue between European patient organisations, and co-ordinate and pool research efforts and resources.
All the SMA Europe representatives are affected by SMA or are parents of affected children, and have personal experience of its effects on patients and their families.
Since SMA Europe’s inception, there has been a determination to combine resources in Europe in order to fund the best researchers on SMA worldwide. With the help of its Scientific Advisory Board, which includes some of the best international experts on neuromuscular diseases, SMA Europe succeeded in launching a successful Call for Projects in 2008, resulting in five new research projects being undertaken internationally. The second annual Call was announced in late March 2009. (See European Call for Projects)
SMA Europe is also seeking ways for organisations in Europe to work together to develop a standardised European patient registry database.
SMA Europe member organisations:
- Asociació Catalana de Enfermetats Neuromusculars (ASEM Catalunya) and Fundame – Spain
- Association Française contre les Myopathies (AFM) – France www.afm-telethon.fr
- Deutsche Gesellschaft fuer Muskelkranke/Initiative SMA (DGM) – Germany
- Famiglie SMA – Italy
- The SMA Trust – United Kingdom
- Vereniging Spierziekten Nederland (VSN) – The Netherlands
- See the www.sma-europe.eu for links to the individual organisations.
Links to SMA Organisations:
FAMILIES OF SMA
Families of Spinal Muscular Atrophy is an international organization dedicated solely to:
- Eradicating spinal muscular atrophy (SMA) by promoting and supporting research.
- Helping families cope with SMA through informational programs and support.
- Educating the public and professional community about SMA.
Funds are specifically directed to scientific, educational or literary purposes in keeping with a charitable organization. Today we have 24 Chapters throughout the United States and over 55,000 members and supporters. We are a collaborative organization where families and friends and researchers are all working together towards the same goal.FSMA organises an annual conference for families and the research community. See their website for dates and details.
FSMA regularly reports on the status of current research into SMA, on their website.
Fight SMA is an international nonprofit organization dedicated to finding a treatment or cure for spinal muscular atrophy (SMA). The organization pursues this objective by raising awareness and funding for SMA research through its international researcher, clinician and family conference, THE GOOD FIGHT, as well as an interactive online community, podcasts, and events.
In addition, FightSMA offers Fighter Mom™, a program for ANY mom who has a child with ANY disease or disorder (www.fightermom.org)
US government agency concerned with SMA
NATIONAL INSTITUTES OF HEALTH
The National Institutes of Health. Extensive information on US medical research. The organization includes The National Institute of Neurological Disorders and Stroke (NINDS), which is the nation’s primary funder of research on the brain and nervous system. Information about SMA and other neurological disorders can be found on the NINDS website: www.ninds.nih.gov/disorders/sma/sma.htm.
THE SMA PATIENT REGISTRY
The SMA Patient Registry. Indiana University’s patient registry programme provides a resource by which individuals and families affected by SMA, and researchers interested in studying SMA, can be in contact with one another. The joining forms can be downloaded from the website.
If you wish to speak to a member of the JTSMA staff directly then you can ring on 0870 774 3651.
TREAT-NMD is an international initiative bringing together twenty-one partner organisations, including doctors, researchers, charities and pharmaceutical companies across Europe which are already working on neuromuscular diseases. This pan-European 'network of excellence' aims at improving treatment and finding cures for patients with neuromuscular disorders.
Visit the website to join the TREAT-NMD patient registry. When a clinical trial is being planned, it is important that patients suitable for that trial can be found and contacted quickly. The best way of ensuring this can happen is to make sure that patients' details are all collected together in a single database or "registry" that contains all the information that researchers will need, including each patient's particular genetic defect and other key information about their disease.
Three of TREAT-NMD most important aims are to:
- Develop common standards for diagnosis and care of neuromuscular disorders
- Establish a Clinical Trials Coordination Centre which includes a patient registry
- Work with industry to develop new and effective treatments for neuromuscular diseases
The TREAT-NMD Newsletter is highly recommended. Register on their website to receive it.
The Italian Famiglie SMA association is composed and managed exclusively by parents of children and adults affected by SMA. Created in 2001, the Association has a membership of hundreds of families, becoming a reference in Italy and abroad for people concerned with SMA.
The goals of Famiglie SMA are:
To inform SMA families on scientific research, on therapies, on assistance that public services can provide.
To promote and finance scientific research on SMA. Famiglie SMA’s Scientific Committee includes some major Italian and worldwide experts, and works with some of the best children’s hospitals and university research centres in Italy.
To promote clinical trials and support the participation of Italian research centres in clinical trials promoted in other countries.
The FSMA website gives update information on the status of SMA Research.
SMA ASSOCIATION OF AUSTRALIA
The Spinal Muscular Atrophy Association of Australia and the SMA Australia website have been set up to promote awareness of SMA and to provide support, information, and equipment to SMA families in Australia. The SMA Association also helps to promote and fund care options, possible treatments and research.
FAMILIES OF SMA HONG KONG
The Hong Kong chapter of the US based organisation – Families of SMA. Now celebrating its 10th year. Fun website with lots of information – and music. Organisation set up by an SMA mother, Mei Ling Fok, and primarily involved with care for SMA sufferers and their families. It employs ten SMA young adults to help run it. Her aim is to build a platform for SMA friends to help SMA friends and all work towards a cure.
Then she can retire!
CHILDREN WITH SMA
Children with SMA
Website providing support and information for those affected by SMA in the Ukraine.
Special Effect is a UK charity dedicated to helping young people with disabilities to enjoy computer games. Many of those affected by SMA need specialist assistance which Special Effect is able to provide.