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The Cure SMA drug pipeline is one of the primary ways we evaluate the success of our research program. The pipeline identifies the major drug programs in development, and tracks their progress from basic research through clinical trials.

Key Measurements

The pipeline measures the progress of our research in two key ways. First, by tracking each individual program all the way to FDA approval. Second, by tracking how these programs are spread among the different therapeutic approaches.

Progress of Individual Drug Programs

Cure SMA's research model funds drug programs at all stages of development. Our basic research program studies the biology and causes of SMA, often revealing new and more effective ways of making drugs. These basic research ideas are then converted into practical drug candidates through drug discovery. Finally, those drug candidates move through the clinical trial process. Our drug pipeline monitors each individual program as it moves through these stages.

First-Ever Approved Therapy for SMA

On December 23, 2016, the FDA announced that it had approved Spinraza, a treatment developed by Biogen and Ionis, making it the first-ever approved therapy for SMA. Cure SMA provided the very first research funding for this program beginning in 2003. The drug pipeline also tracks the implementation of this and other therapies as they are approved.

Balance of Therapeutic Approaches

In order to find a treatment and cure for SMA, we know it's crucial to attack SMA from all sides. As with all scientific research, it's difficult to predict which SMA drug programs might be successful. By investing in a diversity of approaches, we maximize our chances for success. If one drug candidate or one approach fails, we have others to take its place.

The Cure SMA drug pipeline identifies four possible treatment targets:

  • Replacement or correction of the faulty SMN1 gene.
  • Modulation of the low functioning SMN2 “back-up gene.”
  • Neuroprotection of the motor neurons affected by loss of SMN protein.
  • Muscle protection to prevent or restore the loss of muscle function in SMA. 

The therapeutic approaches section of our website describes each of these approaches in more detail.

Research News

We regularly publish updates on SMA research in our news section. Or, sign up for our email list using the box below, and we'll deliver research updates straight to your inbox.

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