Evidence map›Paper›PMID 31793149›Full record

ArticleEMBO molecular medicine2020

Funding community collaboration to develop effective therapies for neurofibromatosis type 1 tumors.

Salvatore La Rosa, Vidya Browder, Annette C Bakker, Jaishri O Blakeley, Sharad K Verma, Ling M Wong, Jill Morris, Naba Bora

Abstract read
In one paragraph

Article in EMBO molecular medicine, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing 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

4 citing papers in PubMed.

  1. Review
  2. Article
  3. Article
  4. 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

8 authors.

Salvatore La RosaChildren's Tumor Foundation, New York, NY, USA.ORCID 0000-0003-2010-4328
Vidya BrowderChildren's Tumor Foundation, New York, NY, USA.
Annette C BakkerChildren's Tumor Foundation, New York, NY, USA.
Jaishri O BlakeleyJohns Hopkins University, Baltimore, MD, USA.
Sharad K VermaJohns Hopkins University, Baltimore, MD, USA.
Ling M WongNational Institute of Neurological Disorders and Stroke, Bethesda, MD, USA.ORCID 0000-0002-6045-5344
Jill MorrisNational Institute of Neurological Disorders and Stroke, Bethesda, MD, USA.
Naba BoraCongressionally Directed Medical Research Programs, Fort Detrick, MD, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

The time from identifying a drug target to a new drug approval is often measured in decades and can take even longer for therapies to treat rare diseases. In fact, 95% of rare diseases do not have a specific therapy approved at all. Coordinated efforts to augment the drug development pipeline along with long-term and comprehensive support that enable scientific breakthroughs for rare diseases are possible, but it requires integration across multiple stakeholders. This article analyzes the coordinated funding efforts of four federal and philanthropic organizations to advance drug development for neurofibromatosis type 1-associated tumors and discusses how these organizations have been collaborating and evolved practices to optimize funding and research support.

Indexed as

Fund RaisingNeurofibromatosis 1HumansRare DiseasesResearch Support as Topic

Identifiers

PMID31793149
PMCPMC6949486

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.