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,1 the principal organ
affected by SMA2,3

After decades of industry research into myostatin inhibition, there is now a different path to treating SMA1,5
Pre-2016
Available treatments:
No treatments
available
2016-2025
Available treatments:
SMN2-targeted treatments6
2026 & Beyond
Available treatments:
ISEMBYLD with an SMN2-targeted treatment1,6
BOTH ACTIONS ARE NOW ACHIEVABLE
Decreasing myostatin activity with ISEMBYLD while increasing SMN protein with an SMN2-targeted treatment1,6
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.
