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Paper · 39201450

Impact of Disease Severity and Disease-Modifying Therapies on Myostatin Levels in SMA Patients.

Mackels L and 4 others. Int J Mol Sci, 2024. Cited by 8 papers, by iCite.

11 sourcespapers-2026-11-12-0.1.0

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ConductScience Library. Impact of Disease Severity and Disease-Modifying Therapies on Myostatin Levels in SMA Patients. Papers, record 39201450. Release papers-2026-11-12-0.1.0, 12 Nov 2026. https://conductscience.org/library/papers/39201450

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Paper

PubMed's record of the paper, with iCite's citation figures. No abstract is shown.

Title
Impact of Disease Severity and Disease-Modifying Therapies on Myostatin Levels in SMA Patients.
PubMed
Journal
Int J Mol Sci
PubMed
Year
2024
PubMed
Publication types
Journal Article
PubMed
Citations, by iCite
8
iCite
Relative citation ratio, by iCite
1.44, provisional
iCite
Revised in PubMed
2024-09-03
PubMed

Authors5

The authors in the order PubMed's record lists them, with the ORCID iDs it states.

OrderAuthorForenameKindORCID iD
1Mackels LLauranehttps://orcid.org/0000-0002-2132-3736
2Mariot VVirginiehttps://orcid.org/0000-0002-3222-9286
3Buscemi LLaura
4Servais LLaurenthttps://orcid.org/0000-0001-9270-4061
5Dumonceaux JJuliehttps://orcid.org/0000-0002-2052-9361

Source PubMed

Topics13

NLM's MeSH indexing of the paper, its major topics first.

MeSH termMajor
MyostatinMajor topic
Muscular Atrophy, SpinalMajor topic
FollistatinMajor topic
Severity of Illness IndexMajor topic
Humans
Male
Female
Oligonucleotides
Retrospective Studies
Child, Preschool
Child
Infant
Adolescent

Source PubMed

Grants0

Each grant PubMed's record writes, matched to the NIH project NIH's grant and paper links name for this paper, and each of those links the record does not write.

None in this release.

Trials0

The trials whose ClinicalTrials.gov record lists the paper, and the trial numbers the paper's PubMed record names.

None in this release.

Comments and corrections0

PubMed's comments and corrections, NLM's own indexing: retractions first, then expressions of concern, errata and updates, and comments last.

None in this release.

References71

The papers the reference list in PubMed's record names, those in our copy of PubMed, in the list's order.

OrderPMIDTitleJournalYearIn PapersSource
127,462,804Myostatin inhibitor ACE-031 treatment of ambulatory boys with Duchenne muscular dystrophy: Results of a randomized, placebo-controlled clinical trial.Muscle Nerve2017https://conductscience.org/library/papers/27462804PubMed
212,459,784Functional improvement of dystrophic muscle by myostatin blockade.Nature2002https://conductscience.org/library/papers/12459784PubMed
327,870,893Activin Receptor Type IIB Inhibition Improves Muscle Phenotype and Function in a Mouse Model of Spinal Muscular Atrophy.PLoS One2016https://conductscience.org/library/papers/27870893PubMed
427,153,267No longer going to waste.Nat Biotechnol2016https://conductscience.org/library/papers/27153267PubMed
532,392,694New and Developing Therapies in Spinal Muscular Atrophy: From Genotype to Phenotype to Treatment and Where Do We Stand?Int J Mol Sci2020https://conductscience.org/library/papers/32392694PubMed
636,846,472Longitudinal developmental profile of newborns and toddlers treated for spinal muscular atrophy.Ther Adv Neurol Disord2023https://conductscience.org/library/papers/36846472PubMed
712,194,980The myostatin propeptide and the follistatin-related gene are inhibitory binding proteins of myostatin in normal serum.J Biol Chem2002https://conductscience.org/library/papers/12194980PubMed
825,322,757A phase 1/2a follistatin gene therapy trial for becker muscular dystrophy.Mol Ther2015https://conductscience.org/library/papers/25322757PubMed
936,849,458Longitudinal Assessment of Creatine Kinase, Creatine/Creatinineratio, and Myostatin as Monitoring Biomarkers in Becker Muscular Dystrophy.Neurology2023https://conductscience.org/library/papers/36849458PubMed
1026,758,873Pharmacologically induced mouse model of adult spinal muscular atrophy to evaluate effectiveness of therapeutics after disease onset.Hum Mol Genet2016https://conductscience.org/library/papers/26758873PubMed

1 to 10 of 71

See all 71 references

Cited by6

The papers in our copy of PubMed whose reference list names this one, newest first. iCite, which reads more sources, counts 8 citations.

PMIDTitleJournalYearIn PapersSource
42,929,233Myostatin/Smad3 in Spinal Muscular Atrophy: Biomarkers of Disease Severity and Treatment Response.Ann Clin Transl Neurol2026https://conductscience.org/library/papers/42929233PubMed
42,310,398Ten years of disease-modifying therapy in spinal muscular atrophy: lessons learned and future directions.Nat Rev Neurol2026https://conductscience.org/library/papers/42310398PubMed
40,565,321Myostatin Modulation in Spinal Muscular Atrophy: A Systematic Review of Preclinical and Clinical Evidence.Int J Mol Sci2025https://conductscience.org/library/papers/40565321PubMed
40,487,335Reconceiving orthopaedic spinal braces for spinal muscular atrophy treatment: A digital modeling and 3D printing combined framework.J Orthop2025https://conductscience.org/library/papers/40487335PubMed
40,368,588Myostatin Levels in SMA Following Disease-Modifying Treatments: A Multi-Center Study.Ann Clin Transl Neurol2025https://conductscience.org/library/papers/40368588PubMed
39,408,601Taldefgrobep Alfa and the Phase 3 RESILIENT Trial in Spinal Muscular Atrophy.Int J Mol Sci2024https://conductscience.org/library/papers/39408601PubMed

Genes2

The genes the Gene search release ties to the paper: Swiss-Prot's references first, then PubTator's text mining.

GeneNCBI Gene IDLinked byIn GenesSource
FST10,468PubTator's text mininghttps://conductscience.org/library/genes/10468NCBI Gene
MSTN2,660PubTator's text mininghttps://conductscience.org/library/genes/2660NCBI Gene

Drugs0

The drugs whose trials, in the Pipelines release, list the paper.

None in this release.

Data0

The data bank entries PubMed's record names. Trial numbers are under Trials.

None in this release.

Data dates

The date of each source this release read.

PubMed
2026-11-06
PubMed
iCite
2026-11-05
iCite
PMC's ID file
2026-11-05
PMC's ID file
NIH RePORTER's grant and paper links
2026-11-01
NIH RePORTER's grant and paper links
Trials
2026-11-12
Gene search
2026-11-11
Pipelines
2026-09-18

Sources 11

  • ChEMBL, through Open TargetsCC BY-SA 3.0
  • ClinicalTrials.govNo license stated
  • HGNCCC0
  • iCiteCC BY 4.0
  • NCBI GenePublic domain (US government)
  • NIH RePORTER's grant and paper linksPublic domain
  • Open TargetsCC0 1.0
  • PMC's ID filePublic domain (US government)
  • PubMedNLM's terms
  • PubTator 3Public domain
  • UniProtCC BY 4.0
Each source's terms
  • ChEMBL, through Open TargetsCC BY-SA 3.0, for the parts from ChEMBL. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
  • ClinicalTrials.govnone stated
  • HGNCCC0: https://www.genenames.org/about/license/
  • iCiteCC BY 4.0
  • NCBI GeneNone named for the file. NCBI says information created by or for the US government on its site is in the public domain, and asks that NLM be acknowledged: https://www.ncbi.nlm.nih.gov/home/about/policies/
  • NIH RePORTER's grant and paper linksPublic domain (NIH RePORTER)
  • Open TargetsCC0 1.0. Open Targets asks that users cite its latest paper: Buniello et al. 2025, Open Targets Platform: facilitating therapeutic hypotheses building in drug discovery, Nucleic Acids Research 53(D1):D1467, PMID 39657122. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
  • PMC's ID fileNone named for the file. NCBI says information created by or for the US government on its site is in the public domain, and asks that NLM be acknowledged: https://www.ncbi.nlm.nih.gov/home/about/policies/
  • PubMedNLM's terms
  • PubTator 3Public domain
  • UniProtCC BY 4.0. UniProt's licence file: "All databases and documents in the UniProt FTP directory may be copied and redistributed freely, without advance permission, provided that this copyright statement is reproduced with each copy." UniProt asks that users cite its latest paper: The UniProt Consortium, UniProt: the Universal Protein Knowledgebase in 2025, Nucleic Acids Research 53:D609-D617, https://doi.org/10.1093/nar/gkae1010. Read on 9 Nov 2026 at https://www.uniprot.org/help/license

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

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