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The National Institute of Neurological Disorders and Stroke (NINDS) has established a model translational research program to accelerate the process of developing a safe and effective treatment for SMA -- an inherited motor neuron disease that leads to muscle wasting and, in its most severe form, death. 

To accomplish the goal of rapidly identifying a suitable therapeutic and completing the preclinical research and development (R&D) studies required for submission of an Investigational New Drug (IND) application, the SMA Project Singapore has adopted the methods used in the pharmaceutical industry to carry out drug discovery and development activities according to accepted industry standards.


The SMA Project focuses on compounds that increase expression of the SMN protein, the protein that is deficient in SMA patients. Through four major contracts, the SMA project has the capabilities to carry out industry-style pre-clinical drug development. These development efforts include:

  • medicinal chemistry
  • in vitro testing
  • pharmacological and toxicological studies
  • animal model testing

The coordinated activities of this multi-site pharmaceutical organization led to the recent filing of a provisional patent application for new indoprofen analogs only 4 months after the start of compound testing. Additional lead compounds are in development or under consideration.


Individuals from organizations engaged in therapeutics development for neurodegenerative and other disorders are encouraged to explore this website and to sign up for the SMA Project ListServ. ListServ members will receive e-mail notification of new solicitations, opportunities to collaborate, notices of the availability of new models and tools, and updates of SMA Project progress. Join our mailing list today.


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Last updated: February 18, 2009 Via SMA Project Singapore