ISEMBYLD is the first and only treatment to directly target
MUSCLE, the principal organ
affected by SMA

ISEMBYLD is the first and only treatment to directly target
MUSCLE,¹the principal organ
affected by SMA^2,3

Illustration of muscle, the principal organ affected by SMA, targeted by ISEMBYLD

After decades of industry research into myostatin inhibition, there is now a different path to treating SMA^1,5

Pre-2016

Available treatments:

No treatments
available

Myostatin signaling
continues

2016-2025

Available treatments:

SMN2-targeted treatments⁶

Myostatin signaling
continues

2026 & Beyond

Available treatments:

ISEMBYLD with an SMN2-targeted treatment^1,6

Myostatin signaling
decreases

BOTH ACTIONS ARE NOW ACHIEVABLE

Decreasing myostatin activity with ISEMBYLD while increasing SMN protein with an SMN2-targeted treatment^1,6

EFFICACY

ISEMBYLD was studied in the pivotal phase 3 SAPPHIRE study.¹ Explore the efficacy data.

RELATED FAQs

What is ISEMBYLD?

ISEMBYLD is a recombinant monoclonal antibody targeting proforms of myostatin, indicated for the treatment of SMA in adults and pediatric patients 2 years of age and older who are currently receiving an SMN2-targeted treatment.¹

How does ISEMBYLD work?

ISEMBYLD inhibits the activation of myostatin (a protein predominantly expressed in skeletal muscle) which is responsible for inhibiting muscle growth, blocking myostatin signaling.¹

How is ISEMBYLD used with SMN2-targeted treatment?

ISEMBYLD is indicated to be used with SMN2-targeted treatments for SMA in adults and pediatric patients 2 years of age and older.¹

References:
1. ISEMBYLD. Prescribing information. Scholar Rock, Inc; 2026. 2. Mercuri E, Finkel RS, Muntoni F, et al. Diagnosis and management of spinal muscular atrophy: Part 1: recommendations for diagnosis, rehabilitation, orthopedic and nutritional care. Neuromuscul Disord. 2018;28(2):103-115. 3. Finkel RS, Crawford TO, Darras BT, et al. Advancing treatment of spinal muscular atrophy through inhibition of the myostatin signaling pathway. Expert Rev Neurother. 2026;26(3):211-225. 4. Antonaci L, Pera MC, Mercuri E. New therapies for spinal muscular atrophy: where we stand and what is next. Euro J Pediatr. 2023;182:2935-2942. 5. Lee SJ. Targeting the myostatin signaling pathway to treat muscle loss and metabolic dysfunction. J Clin Invest. 2021;131(9):e148372. 6. Day JW, Howell K, Place A, et al. Advances and limitations for the treatment of spinal muscular atrophy. BMC Pediatr. 2022;22(1):632.

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ISEMBYLD is the first and only treatment to directly target
MUSCLE, the principal organ
affected by SMA

After decades of industry research into myostatin inhibition, there is now a different path to treating SMA^1,5

ISEMBYLD was studied in the pivotal phase 3 SAPPHIRE study.¹ Explore the efficacy data.

Indication

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS

Fractures

ADVERSE REACTIONS

USE IN SPECIFIC POPULATIONS

Pregnancy and Lactation

Females and Males of Reproductive Potential

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ISEMBYLD is the first and only treatment to directly target MUSCLE, the principal organ affected by SMA

After decades of industry research into myostatin inhibition, there is now a different path to treating SMA1,5

ISEMBYLD was studied in the pivotal phase 3 SAPPHIRE study.1 Explore the efficacy data.

CONNECT WITH US

ISEMBYLD is the first and only treatment to directly target
MUSCLE, the principal organ
affected by SMA

After decades of industry research into myostatin inhibition, there is now a different path to treating SMA^1,5

ISEMBYLD was studied in the pivotal phase 3 SAPPHIRE study.¹ Explore the efficacy data.