Cure SMA Releases Updated Drug Pipeline

We’ve recently released an update to the SMA drug pipeline. This latest version includes:

  • 20 active programs, including three approved therapies.
  • 10 pharmaceutical partners.
  • 12 programs in clinical trials.
  • An ever-increasing breadth of potential treatment approaches to SMA.

Effective Treatments for All Ages, Stages and Types of SMA

The updated version of this pipeline reflects our community’s progress in gaining access to Spinraza, Zolgensma, and Evrysdi, as well as the progress of 17 additional programs currently in development.

There are many systems, pathways and processes that are affected in SMA, and there may be additional ways to treat SMA that work on these other areas—collectively referred to as “non-SMN” approaches. These non-SMN approaches include drugs that work on the muscles and nerves.

Research strongly suggests that a combination of SMN-enhancing and non-SMN approaches is the best route to treat all ages, stages, and types of SMA. This allows us to attack SMA from all sides and ensures that comprehensive, effective treatments are available. Because of this, Cure SMA’s strategic research model is focused on the complementary goals of increasing the total number of programs in the drug pipeline and increasing the diversity of programs in the pipeline.

There are many clinical trials in progress for both SMN-enhancing and non-SMN approaches including trials:

  • Investigating optimization of FDA approved SMN-enhancing therapies.
  • Investigating novel drug candidates focused on non-SMN targets.
  • Investigating investigational non-SMN targeted drugs in combination with approved SMN-enhancing therapies.

Balance of Therapeutic Approaches

In order to find more treatments and a 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:

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

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

Thank You

The dedicated support of our community has made all of this possible. Whether families raising funds and awareness, researchers investigating new potential treatments, or pharmaceutical and regulatory partners helping us take the next steps—everyone has a role to play. Thank you for your hard work and dedication.


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