Evidence map›Paper›PMID 40065173›Full record

ArticleNature medicine2025

The Rare Therapies Launchpad: a pilot program for individualized medicines in the UK.

Daniel J O'Connor, Parker Moss, Matthew Wood, Martin Murphy, Michael Parker, Nicola Blackwood, Matthew A Brown, Deb Lancaster, Vanessa Newman, Jenny Taylor and 2 more

Abstract readLetter

PubMed Publisher

In one paragraph

Article in Nature medicine, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers.

0numbers the graph read from it

0cells of the map it votes in

6citing 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

6 citing papers in PubMed.

From N-of-1 therapy to health-system implementation.Nature reviews. Drug discovery · 2026
Article
TrialR: critical enablers and the need for reusable Rare Disease Clinical Trial infrastructure in Western Australia.Orphanet journal of rare diseases · 2026
Article
A baby benefits from personalized gene editing in the clinic.Nature · 2025
Article
The N=1 Collaborative: advancing customized nucleic acid therapies through collaboration and data sharing.Nucleic acids research · 2025
Review
Novel advanced patient-derivedFrontiers in neurology
Review
From roadmap to a sustainable end-to-end individualized therapy pathway.Therapeutic advances in rare disease
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

12 authors.

Daniel J O'ConnorThe Association of the British Pharmaceutical Industry, London, UK.

Parker MossCancer Research UK, London, UK.

Matthew WoodOxford-Harrington Rare Disease Centre, Department of Paediatrics, University of Oxford, Oxford, UK.

Martin MurphyRosa Genomic Medicines, London, UK.

Michael ParkerUniversity of Oxford, Oxford, UK.ORCID http://orcid.org/0000-0002-7054-4711

Nicola BlackwoodGenomics England, London, UK.

Matthew A BrownGenomics England, London, UK.ORCID http://orcid.org/0000-0003-0538-8211

Deb LancasterGenomics England, London, UK.

Vanessa NewmanGenomics England, London, UK.

Jenny TaylorCentre for Human Genetics and Oxford NIHR Biomedical Research Centre, University of Oxford, Oxford, UK.ORCID http://orcid.org/0000-0003-3602-5704

Tim YuBoston Children's Hospital, Boston, MA, USA.ORCID http://orcid.org/0000-0003-2988-7701

Julia VitarelloMila's Miracle Foundation, Boulder, CO, USA. julia@stopbatten.org.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

PubMed holds no abstract for this paper.

Identifiers

PMID40065173

DOI10.1038/s41591-025-03547-4

What OpenQuestion holds

Textmetadata

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.