ACE-031
ACE-031 (ramatercept) is a lab-made fusion protein: the outer part of the human activin receptor type IIB joined to the Fc stem of a human antibody. It works as a decoy that soaks up myostatin and related signals that limit muscle growth. It was developed as a possible treatment for Duchenne muscular dystrophy, but development stopped after safety concerns in a trial in boys.
Approval status
Not approved anywhere. A phase 2 trial in boys with Duchenne muscular dystrophy was stopped early in 2011 because of nosebleeds and small dilated blood vessels, and development was halted. It is still sold on the black market as a research chemical.12478
What it’s used or studied for
- Human studies
Tested in boys with Duchenne muscular dystrophy: trends toward more lean mass and steadier walking distance, but the trial was stopped for safety reasons.12
- Human studies
A single injection increased lean body mass and thigh muscle volume in healthy postmenopausal women.3
- Animal & lab only
Increased body weight, muscle weight and muscle fiber size in mice.6
- Animal & lab only
Increased lean mass, muscle fiber size and muscle force in marmoset monkeys.5
Each label shows how strong the evidence is: an approved use, human studies, animal or lab studies only, or a popular claim with no published studies. Being studied is not the same as being proven safe or effective.
Side effects
Common
- Nosebleeds
- Small dilated blood vessels in the skin (telangiectasia)
- Redness at the injection site
- Headache
In the Duchenne trial, nosebleeds and telangiectasia led to the study being stopped, although no events were rated serious or severe. Later papers also mention gum bleeding, which the trial's posted results do not list, and link these bleeding effects to blocking BMP9, a signal that maintains blood vessels. A single-dose study in healthy women also lowered FSH, a reproductive hormone.12345
Cautions
- A doping lab found that black-market "ACE-031" was not ACE-031: 12 of 14 products held the full-length receptor instead, plus many other proteins, and 2 held no receptor at all.7
- The published human data are short-term: a single-dose study in healthy women and a trial in boys that was stopped early. There are no long-term safety data.13
Sources
- Campbell C et al. Myostatin inhibitor ACE-031 treatment of ambulatory boys with Duchenne muscular dystrophy (Muscle Nerve, 2017)
- ClinicalTrials.gov NCT01099761: Study of ACE-031 in subjects with Duchenne muscular dystrophy (terminated; results)
- Attie KM et al. A single ascending-dose study of muscle regulator ACE-031 in healthy volunteers (Muscle Nerve, 2013)
- Suh J, Lee YS. Myostatin inhibitors: panacea or predicament for musculoskeletal disorders? (J Bone Metab, 2020)
- Cadena SM et al. ACE-031 increases muscle mass and strength in the common marmoset (PLoS One, 2026)
- Cadena SM et al. A soluble activin type IIB receptor promotes skeletal muscle growth independent of fiber type (J Appl Physiol, 2010)
- Reichel C et al. Gel electrophoretic detection of black market ACE-031 (Drug Test Anal, 2025)
- WADA 2026 Prohibited List
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