In a small study, boys with Duchenne muscular dystrophy received injections of a drug called ACE-031 every few weeks. The drug did not cause serious side effects, but the study was stopped early because some boys developed nosebleeds and small red spots on the skin, which are safety concerns.
See the scientific wording
Subcutaneous administration of the myostatin inhibitor ACE-031 every 2-4 weeks in ambulatory boys with Duchenne muscular dystrophy was not associated with serious or severe adverse events in a small early-terminated randomized controlled trial; however, the study was terminated due to epistaxis and telangiectasias, indicating a potential safety signal.
Strong evidence
Randomized trialsOne moderate-quality study supports this claim, so treat this as an early signal rather than settled science.
What the research says
1 study reviewedSupporting (1)
Randomized Controlled TrialHuman2017
The study found that the drug didn't cause serious side effects, but because some boys had nosebleeds and small red spots, they stopped the study early. That's exactly what the claim says.
Contradicting (0)
No contradicting studies found yet
That doesn't mean it's settled — it just means no study has tested the opposite.
Quality-weighted scoring: we follow the GRADE framework — each study is rated High, Moderate, Low, or Very Low based on study design, methodology rigor, and risk of bias. A single high-quality RCT can outweigh several weaker observational studies.
Scores reflect study quality, not just count.
ACE-031 is a drug that sticks to a protein called myostatin, which normally stops muscles from growing. By blocking myostatin, ACE-031 lets muscles grow bigger and stronger. But myostatin-like proteins also help keep blood vessels healthy and strong. When ACE-031 also blocks those, it can make blood vessels weak and leaky, causing nosebleeds and red spots on the skin.
Score breakdown, mechanism chain, raw evidence, ideal studies needed & 1 supporting study
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In a small study, boys with Duchenne muscular dystrophy received injections of a drug called ACE-031 every few weeks. The drug did not cause serious side effects, but the study was stopped early because some boys developed nosebleeds and small red spots on the skin, which are safety concerns.
Mechanism
1 studyACE-031 blocks a protein that stops muscle growth, so muscles can grow. But it also blocks proteins that keep blood vessels healthy, which can cause nosebleeds and red spots. The drug did not cause serious side effects, but these blood vessel problems led to stopping the study early.
ACE-031 is a drug that sticks to a protein called myostatin, which normally stops muscles from growing. By blocking myostatin, ACE-031 lets muscles grow bigger and stronger. But myostatin-like proteins also help keep blood vessels healthy and strong. When ACE-031 also blocks those, it can make blood vessels weak and leaky, causing nosebleeds and red spots on the skin.
ACE-031 binds to myostatin and related ligands such as activin A and GDF-11 in the circulation and extracellular space.
Binding of ACE-031 prevents these ligands from activating native activin receptor type IIB (ActRIIB) on muscle cells and other tissues.
Blockade of ActRIIB signaling reduces Smad2/3 phosphorylation and downstream negative regulation of muscle growth.
Increased muscle protein synthesis and myogenesis lead to gain in lean mass and potential improvement in muscle function.
Reduced adiposity and increased bone mineral density occur as secondary effects of altered growth factor signaling.
Inhibition of ligands like activin A and GDF-11 may disrupt endothelial integrity and angiogenesis, leading to vascular fragility manifesting as epistaxis and telangiectasias.
Evidence from Studies
Supporting (1)
Community contributions welcome
Myostatin inhibitor ACE‐031 treatment of ambulatory boys with Duchenne muscular dystrophy: Results of a randomized, placebo‐controlled clinical trial
The study found that the drug didn't cause serious side effects, but because some boys had nosebleeds and small red spots, they stopped the study early. That's exactly what the claim says.
Contradicting (0)
Community contributions welcome
Score Breakdown
No multi-axis breakdown available yet. The overall Pro / Against score above is the best signal.
- No clinical evidence is available; the score reflects mechanistic plausibility only.
What Would Prove This
Per GRADE and EBM methodology, here is what ideal scientific evidence would look like to definitively prove or disprove this claim, ordered from strongest to weakest.
Systematic Review of Myostatin Inhibitors in Duchenne Muscular Dystrophy: Safety and Adverse Events
Systematic review with meta-analysis of RCTs and observational studies on myostatin inhibitors (e.g., ACE-031) in DMD patients, focusing on adverse events including epistaxis and telangiectasias, with subgroup analysis by age and ambulatory status.
Double-Blind, Placebo-Controlled Trial of ACE-031 in Ambulatory Boys with Duchenne Muscular Dystrophy: Long-Term Safety and Efficacy
Multicenter, double-blind, placebo-controlled RCT in ambulatory boys with DMD (age 5-12), randomized to subcutaneous ACE-031 every 2-4 weeks vs placebo for 12 months, with primary outcome of incidence of serious adverse events and secondary outcomes including epistaxis and telangiectasias.
Long-Term Safety Monitoring Cohort of Duchenne Muscular Dystrophy Patients Treated with Myostatin Inhibitors
Prospective cohort study of DMD patients receiving myostatin inhibitors (including ACE-031) from multiple centers, followed for at least 2 years with regular adverse event monitoring and comparison to a matched untreated cohort.
Case-Control Study of Epistaxis and Telangiectasias in Duchenne Muscular Dystrophy Patients Exposed to ACE-031
Nested case-control within a cohort of DMD patients: cases are those who developed epistaxis/telangiectasias, controls are those without, matched by age and disease severity, with exposure to ACE-031 assessed via medical records.
Case Series of Epistaxis and Telangiectasias in Duchenne Muscular Dystrophy Patients Treated with ACE-031
Retrospective or prospective collection of case reports of DMD patients who developed epistaxis and/or telangiectasias while on ACE-031, including detailed clinical history, dosage, timing, and outcomes.