Evidence map›Paper›PMID 41552386›Full record

ArticleMolecular therapy. Nucleic acids2026

PPMO-based exon skipping therapy improves respiratory function in the

Debolina D Biswas, Maran Y Hernandez Rodriguez, Lea El Haddad, Gwendolyn Hoffmann, Elena Copson, Sloane Mayman, Julia Jose, Sydney Lang, Olivia Chen, Andrea Dela Pena and 4 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy. Nucleic acids, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
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

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

14 authors.

Debolina D BiswasDivision of Pulmonary and Sleep Medicine, Department of Pediatrics, Duke University Medical Center, Box 2644, Durham, NC 27710, USA.
Maran Y Hernandez RodriguezDivision of Pulmonary and Sleep Medicine, Department of Pediatrics, Duke University Medical Center, Box 2644, Durham, NC 27710, USA.
Lea El HaddadDivision of Pulmonary and Sleep Medicine, Department of Pediatrics, Duke University Medical Center, Box 2644, Durham, NC 27710, USA.
Gwendolyn HoffmannSarepta Therapeutics, Inc., 215 First Street, Cambridge, MA 02142, USA.
Elena CopsonSarepta Therapeutics, Inc., 215 First Street, Cambridge, MA 02142, USA.
Sloane MaymanDivision of Pulmonary and Sleep Medicine, Department of Pediatrics, Duke University Medical Center, Box 2644, Durham, NC 27710, USA.
Julia JoseDivision of Pulmonary and Sleep Medicine, Department of Pediatrics, Duke University Medical Center, Box 2644, Durham, NC 27710, USA.
Sydney LangDivision of Pulmonary and Sleep Medicine, Department of Pediatrics, Duke University Medical Center, Box 2644, Durham, NC 27710, USA.
Olivia ChenDivision of Pulmonary and Sleep Medicine, Department of Pediatrics, Duke University Medical Center, Box 2644, Durham, NC 27710, USA.
Andrea Dela PenaDivision of Pulmonary and Sleep Medicine, Department of Pediatrics, Duke University Medical Center, Box 2644, Durham, NC 27710, USA.
Emerson LevinDivision of Pulmonary and Sleep Medicine, Department of Pediatrics, Duke University Medical Center, Box 2644, Durham, NC 27710, USA.
Mariam AbdelbarrDivision of Pulmonary and Sleep Medicine, Department of Pediatrics, Duke University Medical Center, Box 2644, Durham, NC 27710, USA.
Aoife SlyneDivision of Pulmonary and Sleep Medicine, Department of Pediatrics, Duke University Medical Center, Box 2644, Durham, NC 27710, USA.
Mai K ElMallahDivision of Pulmonary and Sleep Medicine, Department of Pediatrics, Duke University Medical Center, Box 2644, Durham, NC 27710, USA.

Funding

Optimizing Gene Therapy for Respiratory Insufficiency in Duchenne Muscular DystrophyR01HL171282 · NHLBI · DUKE UNIVERSITY · PI Mai ElMallah · 2024 to 2026
$1.9M
NHLBI NIH HHS R01 HL171282
6 · The paper itself

Abstract

Duchenne muscular dystrophy (DMD) is a severe X-linked recessive disorder caused by a deficiency of dystrophin, leading to progressive muscle degeneration and eventually cardiorespiratory failure. Exon-skipping therapies using cell-penetrating peptide-conjugated phosphorodiamidate morpholino oligomers (PPMOs) restore production of a shortened but functional dystrophin protein. Since respiratory insufficiency is the leading cause of morbidity and mortality in DMD, we sought to examine the impact of PPMO on respiratory pathology. We evaluated the effects of RC-1001, a PPMO targeting a dystrophin mutation, in

Indexed as

breathingDuchenne Muscular DystrophyDystrophinexon skipping therapyFlexiVentforced oscillometryMT: Oligonucleotides: Therapies and Applicationspeptide-conjugated phosphorodiamidate morpholino oligomersPPMOrespiratory dysfunctionwhole body plethysmography

Identifiers

PMID41552386
PMCPMC12809079

What Socratic holds

Textmetadata
LicenceCC BY-NC-ND
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 Socratic graph.