Evidence map›Paper›PMID 31408013›Full record

ReviewCurrent opinion in neurology2019

Advances in primary mitochondrial myopathies.

Isabella Peixoto de Barcelos, Valentina Emmanuele, Michio Hirano

Abstract readReview
In one paragraph

Review in Current opinion in neurology, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 30 papers.

0numbers the graph read from it
0cells of the map it votes in
30citing papers in PubMed
1.4field-weighted citation impact, top 19% 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

30 citing papers in PubMed, 43 citations in OpenAlex.

  1. Trial
  2. Progress on cell therapy for skeletal muscle disorders.Advanced drug delivery reviews · 2026
    Review
  3. Review
  4. Article
  5. Article
  6. Article
  7. Observational
  8. Review
  9. Article
  10. Article
  11. Review
  12. Review
  13. A clinical approach to diagnosis and management of mitochondrial myopathies.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2024
    Review
  14. Article
  15. Article
  16. Article
  17. Article
  18. Article
  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

3 authors at 1 institution in 1 country.

Isabella Peixoto de BarcelosDepartment of Neurology, Columbia University Irving Medical Center, New York, New York, USA.
Valentina Emmanuele
Michio Hirano
Columbia University Irving Medical Center · US

Funding

The Impact of COVID-19 on People Living with Rare Diseases and Their FamiliesU2CTR002818 · NCATS · CINCINNATI CHILDRENS HOSP MED CTR · PI MICHAEL SEID · 2019 to 2026
$51.2M
The North American Mitochondrial Disease Consortium (NAMDC)U54NS078059 · NINDS · COLUMBIA UNIVERSITY HEALTH SCIENCES · PI HIRANO, MICHIO · 2011 to 2023
$17.5M
Tech-CoreP01HD080642 · NICHD · COLUMBIA UNIVERSITY HEALTH SCIENCES · PI QUINZII, CATARINA M. · 2014 to 2018
$7.8M
Therapeutic potential of enhanced mitochondrial biogenesis for paclitaxel-induced peripheral neuropathyR21CA226672 · NCI · COLUMBIA UNIVERSITY HEALTH SCIENCES · PI HIRANO, MICHIO · 2018 to 2019
$378k
NCATS NIH HHS U2C TR002818NCI NIH HHS R21 CA226672NICHD NIH HHS P01 HD080642NINDS NIH HHS U54 NS078059
6 · The paper itself

Abstract

purpose of reviewAlthough mitochondrial diseases impose a significant functional limitation in the lives of patients, treatment of these conditions has been limited to dietary supplements, exercise, and physical therapy. In the past few years, however, translational medicine has identified potential therapies for these patients. RECENT

findingsFor patients with primary mitochondrial myopathies, preliminary phase I and II multicenter clinical trials of elamipretide indicate safety and suggest improvement in 6-min walk test (6MWT) performance and fatigue scales. In addition, for thymidine kinase 2-deficient (TK2d) myopathy, compassionate-use oral administration of pyrimidine deoxynucleosides have shown preliminary evidence of safety and efficacy in survival of early onset patients and motor functions relative to historical TK2d controls. SUMMARY: The prospects of effective therapies that improve the quality of life for patients with mitochondrial myopathy underscore the necessity for definitive diagnoses natural history studies for better understanding of the diseases.

Indexed as

Quality of LifeClinical Trials as TopicExerciseFatigueHumansMitochondrial MyopathiesOligopeptidesarginyl-2,'6'-dimethyltyrosyl-lysyl-phenylalaninamideOligopeptides

Identifiers

PMID31408013
PMCPMC6938233
OpenAlexW2968878212

What OpenQuestion holds

Textmetadata
LicenceTDM
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.