A New Focus for SMA

Finding a clinically actionable therapeutic target to help address the muscular component of the neuromuscular unit is an important area of focus to advance treatment for people living with SMA.³

Diagram illustrating myostatin binding at the muscle cell membrane

MYOSTATIN: A POTENTIAL TARGET IN SMA

Maintenance of skeletal muscle homeostasis is dependent on a balance between anabolic and catabolic signaling pathways.²⁸

Myostatin, a potent naturally occurring protein predominantly expressed in skeletal muscles, exerts an inhibitory effect on muscle growth and overall muscle size.²²

By targeting myostatin, it may be possible to promote myogenesis and protein synthesis while suppressing muscle catabolism.²⁹

Learn more about myostatin as a potential target to address the clinical issues of muscles in people living with SMA

Diana Castro, MD

Episode 3Dec 5, 2024

Myostatin Review with Jena Krueger, MD Leslie Cortes, PhD, and Adam Fogel, PhD

0:000:00

In this episode: New research approaches in spinal muscular atrophy (SMA) are now targeting muscle. In this episode, host Diana Castro, MD, discusses myostatin—a new potential target in SMA. She is joined by Jena Krueger, MD, a pediatric neurologist, and Leslie Cortes, PhD, and Adam Fogel, PhD, researchers from Scholar Rock, to talk about what myostatin is, why it matters in muscle, and why it’s such an important focus for future developments in SMA.

Listen to more EPISODES

For children and adults with neuromuscular diseases, the potential of myostatin inhibition is supported by more than two decades of preclinical and clinical research.^22,29-31

Mckenna, 16

Living with SMA and advocating for progress

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References

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Abati E, Manini A, Comi GP, Corti S. Inhibition of myostatin and related signaling pathways for the treatment of muscle atrophy in motor neuron diseases. Cell Mol Life Sci. 2022;79(7):374.
Day JW, Howell K, Place A, et al. Advances and limitations for the treatment of spinal muscular atrophy. BMC Pediatr. 2022;22(1):632.
Gnazzo M, Pisanò G, Baldini V, et al. Myostatin modulation in spinal muscular atrophy: A systematic review of preclinical and clinical evidence. Int J Mol Sci. 2025;26(12).
Schroth M, Deans J, Arya K, et al. Spinal muscular atrophy update in best practices: recommendations for diagnosis considerations. Neurol Clin Pract. 2024;14(4):e200310.
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Finkel RS, Farrar MA, Saito K, et al. Final Safety and Efficacy Data From the SHINE Study in Participants With Infantile-Onset and Later-Onset SMA. Poster presented at: Annual Cure SMA Research and Clinical Care Meeting; June 2024; Austin, TX.
Servais L, Oskoui M, Day J, et al. SUNFISH Parts 1 and 2: 4-year Efficacy and Safety Data of Risdiplam in Types 2 and 3 Spinal Muscular Atrophy (SMA). Poster presented at: Muscular Dystrophy Association (MDA) Clinical and Scientific Conference; March 2025; Dallas, TX.
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A New Focus for SMA

MYOSTATIN: A POTENTIAL TARGET IN SMA

Myostatin Review with Jena Krueger, MD Leslie Cortes, PhD, and Adam Fogel, PhD

LIFE TAKES MUSCLE,CHANGE TAKES YOU

Welcome!

LIFE TAKES MUSCLE,
CHANGE TAKES YOU