Research Tools
For more information about SMA, the following is a list of resources that may be of use. These websites contain information about other organizations in the SMA community, the biology and genetics of SMA, SMA clinical trials and potential drug treatments, and research-related information for scientists, including available mouse models, licenses, SMA patents and antibodies.Animal Models:
Licenses:
The SMA Foundation has entered into licensing agreements with several Institutions in order to facilitate access to critical research tools, while giving the Inventors and Institutions acknowledgment of key contributions and fair compensation for their intellectual property. The Foundation offers the opportunity to obtain sublicenses for the research and therapeutics development tools listed below. The portfolio is designed to reduce or eliminate barriers for drug discovery efforts and to accelerate the development of a treatment for SMA. For more information, please contact us at This e-mail address is being protected from spam bots, you need JavaScript enabled to view it
- University of Wuerzburg
- Certain SMA mouse models deficient in Smn mRNA and protein due to the targeted mutation Smntm1Msd and generated in the laboratory of Dr. Michael Sendtner. The allele has been additionally incorporated into several mouse strains as below.
- The Ohio State University
- Certain SMA mouse models that are deficient in mouse SMN mRNA and protein as above, but including human SMN transgenes as generated in the laboratory of Dr. Arthur Burghes. Strain designations follow.
- Mouse strains referred to by the University of Wuerzburg and The Ohio State University licenses are available from The Jackson Laboratories:
- FVB.Cg-Tg(SMN2)89Ahmb Smntm1Msd/J (Stock # 005024)
- FVB.Cg-Tg(SMN2*delta7)4299Ahmb Tg(SMN2)89Ahmb Smntm1Msd/J (Stock # 005025)
- FVB.Cg-Tg(SMN2)89Ahmb Tg(SMN1*A2G)2023Ahmb Smntm1Msd/J (Stock # 005026)
Patents:
- For a spreadsheet that gives basic information about antibodies used in SMA research, please click here. Reviews and comments on these antibodies have been gathered from SMA researchers by the SMA Foundation.
- If you have any experience with antibodies used in SMA research, we strongly encourage you to submit reviews or comments on their general usefulness and reliability, applications tested and dilution notes. For information on how to provide comments or updates, please email the This e-mail address is being protected from spam bots, you need JavaScript enabled to view it .
Other SMA Community Resources
- Association Francaise contre les Myopathies (France)
- Families of SMA
- Fight SMA
- Initiative SMA (Germany)
- The Jennifer Trust for Spinal Muscular Atrophy (UK)
- Miracle for Madison
- Muscular Dystrophy Association
- Payton’s Pals
- SMA Angels Charity
- SMA Europe
- SMA Support
- The SMA Trust (UK)
- Treat-NMD (Europe)
Other Clinical Resources on SMA:
- National Institute of Neurological Disorders and Stroke (NINDS ) Information Page on SMA
- MedlinePlus Information on SMA
- GeneClinics Clinical Review of SMA
- Prior, TW and Russman, BS, (Updated July 15, 2004). Spinal Muscular Atrophy. In GeneReviews at GeneTests: Medical Genetics Information Resource (Database online). © University of Washington, Seattle. 1997-2005.
- Genetic Testing Resources from GeneTests
- In: GeneTests: Medical Genetics Information Resource (Database online). Educational Materials: Spinal Muscular Atrophy Laboratories offering clinical testing. © University of Washington, Seattle. 1993-2005.
- Columbia University Medical Center Spinal Muscular Atrophy Clinical Research Center General Overview of SMA and Information on SMA Clinical Research
- Treat-NMD Clinical Research Initiatives in Europe
- SMA Foundation Bibliography
- Alzheimer's Research Forum - a website where you can look for research on SMA and other motor neuron and neurodegenerative diseases.
- PubMed - provides abstracts to biomedical literature
- Highwire - Highwire Press, Internet Imprint of the Stanford University Libraries
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For Families: A Guide to Standard of Care Consensus Statement
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