Therapeutics Development

Drug Discovery and Development
A series of advances over the past 15 years has catapulted SMA to the threshold of a treatment or cure. Targets that are being evaluated for the treatment of the disease include: altering the transcription and translation of SMN2, replacing the full-length SMN protein through gene therapy, stabilizing and enhancing the function of existing motor units, and preventing further motor neuron loss.

The compounds that are currently being evaluated in patients have demonstrated an ability to increase SMN2 transcription or improve SMN2 splicing so that more full-length protein is produced. Several of the compounds are drugs that are approved by the FDA for other purposes. There are also many other compounds that are in the early stages of investigation and libraries of compounds are being screened for more potential treatment options.

Information for Industry:
1. For an overview of the current status of compounds that are being evaluated for SMA treatment, please click here.
2. For a copy of our analysis of the marketplace for SMA therapeutics development, please email Cynthia Joyce at This e-mail address is being protected from spam bots, you need JavaScript enabled to view it .
3. For information about SMA animal models, please click here .
4. For information about SMA clinical trials, please visit Clinical Research.