Evidence map›Paper›PMID 31388039›Full record

ArticleScientific reports2019

Follistatin-based ligand trap ACE-083 induces localized hypertrophy of skeletal muscle with functional improvement in models of neuromuscular disease.

R S Pearsall, M V Davies, M Cannell, J Li, J Widrick, A W Mulivor, S Wallner, M E Troy, M Spaits, K Liharska and 6 more

Open access · goldAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Scientific reports, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 33 papers.

0numbers the graph read from it
0cells of the map it votes in
33citing papers in PubMed
2.5field-weighted citation impact, top 10% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

33 citing papers in PubMed, 49 citations in OpenAlex.

  1. Trial
  2. Article
  3. Review
  4. Review
  5. Article
  6. Review
  7. Unravelling the Complexity of Sarcopenia Through a Systems Biology Approach.International journal of molecular sciences · 2025
    Review
  8. Review
  9. Deciphering Facioscapulohumeral Dystrophy in the clinical trials era: where are we now?Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology · 2025
    Review
  10. Review
  11. Review
  12. Muscular Dystrophies.Advances in experimental medicine and biology · 2025
    Review
  13. Review
  14. Review
  15. Review
  16. Sarcopenia in chronic kidney disease: from bench to bedside.The Korean journal of internal medicine · 2023
    Review
  17. Review
  18. Myostatin: A Skeletal Muscle Chalone.Annual review of physiology · 2023
    Review
  19. Article
  20. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

16 authors at 4 institutions in 2 countries.

R S PearsallAcceleron Pharma, Cambridge, MA, USA. spearsall@xlrn.com.
M V DaviesAcceleron Pharma, Cambridge, MA, USA.
M CannellAcceleron Pharma, Cambridge, MA, USA.
J LiAcceleron Pharma, Cambridge, MA, USA.
J WidrickDivision of Genetics and Genomics, The Manton Center for Orphan Disease Research, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.
A W MulivorAcceleron Pharma, Cambridge, MA, USA.
S WallnerAcceleron Pharma, Cambridge, MA, USA.
M E TroyAcceleron Pharma, Cambridge, MA, USA.
M SpaitsAcceleron Pharma, Cambridge, MA, USA.
K LiharskaAcceleron Pharma, Cambridge, MA, USA.
D SakoAcceleron Pharma, Cambridge, MA, USA.
R CastonguayAcceleron Pharma, Cambridge, MA, USA.
S KeatesAcceleron Pharma, Cambridge, MA, USA.
A V GrinbergAcceleron Pharma, Cambridge, MA, USA.
R N V S SuraganiAcceleron Pharma, Cambridge, MA, USA.
R KumarAcceleron Pharma, Cambridge, MA, USA.
Acceleron Pharma (United States) · USHarvard University · USHospital for Sick Children · CATScan Therapeutics (United States) · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Skeletal muscle is under inhibitory homeostatic regulation by multiple ligands of the transforming growth factor-β (TGFβ) superfamily. Follistatin is a secreted protein that promotes muscle growth and function by sequestering these ligands extracellularly. In the present study, we evaluated the potential of ACE-083 - a locally acting, follistatin-based fusion protein - as a novel therapeutic agent for focal or asymmetric myopathies. Characterization of ACE-083 in vitro revealed its high affinity for heparin and extracellular matrix while surface plasmon resonance and cell-based assays confirmed that ACE-083 binds and potently neutralizes myostatin, activin A, activin B and growth differentiation factor 11 (GDF11). Intramuscular administration of ACE-083 caused localized, dose-dependent hypertrophy of the injected muscle in wild-type mice and mouse models of Charcot-Marie-Tooth disease (CMT) and Duchenne muscular dystrophy, with no evidence of systemic muscle effects or endocrine perturbation. Importantly, ACE-083 also increased the force of isometric contraction in situ by the injected tibialis anterior muscle in wild-type mice and disease models and increased ankle dorsiflexion torque in CMT mice. Our results demonstrate the potential of ACE-083 as a therapeutic agent for patients with CMT, muscular dystrophy and other disorders with focal or asymmetric muscle atrophy or weakness.

Indexed as

ActivinsAnimalsBone Morphogenetic ProteinsCharcot-Marie-Tooth DiseaseDisease Models, AnimalFollistatinGrowth Differentiation FactorsHumansHypertrophyLigandsMaleMiceMice, Inbred mdxMuscle, SkeletalMuscle StrengthMuscular Dystrophy, Duchenneactivin Aactivin BActivinsBone Morphogenetic ProteinsFollistatinFST protein, humanGdf11 protein, mouseGrowth Differentiation FactorsLigandsMstn protein, mouseMyostatinReceptors, IgGRecombinant Fusion Proteins

Identifiers

PMID31388039
PMCPMC6684588
OpenAlexW2966592669

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.