Patient organizations and research on rare diseases.

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.

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.

Who cites it

13 citing papers in PubMed.

1. Sorafenib for Advanced and Refractory Desmoid Tumors.The New England journal of medicine · 2018
   Trial
2. Implementing core outcomes in kidney disease: report of the Standardized Outcomes in Nephrology (SONG) implementation workshop.Kidney international · 2018
   Article
3. Common Elements in Rare Kidney Diseases: Conclusions from a Kidney Disease: Improving Global Outcomes (KDIGO) Controversies Conference.Kidney international · 2017
   Article
4. Lymphangioleiomyomatosis: A Monogenic Model of Malignancy.Annual review of medicine · 2017
   Review
5. Initial report of the osteogenesis imperfecta adult natural history initiative.Orphanet journal of rare diseases · 2015
   Article
6. Commentary on Vickers.Clinical trials (London, England) · 2014
   Article
7. Rare inherited kidney diseases: challenges, opportunities, and perspectives.Lancet (London, England) · 2014
   Review
8. Novel molecular pathways in Gorham disease: implications for treatment.Pediatric blood & cancer · 2014
   Review
9. Improving the diagnosis and treatment of CRPS: insights from a clinical immunologist's personal experience with an underrecognized neuroinflammatory disorder.Journal of neuroimmune pharmacology : the official journal of the Society on NeuroImmune Pharmacology · 2013
   Article
10. Making connections: using telehealth to improve the diagnosis and treatment of Complex Regional Pain Syndrome, an underrecognized neuroinflammatory disorder.Journal of neuroimmune pharmacology : the official journal of the Society on NeuroImmune Pharmacology · 2013
    Review
11. Global effort against rare and orphan diseases.European respiratory review : an official journal of the European Respiratory Society · 2012
    Article
12. Caregiver and adult patient perspectives on the importance of a diagnosis of 22q11.2 deletion syndrome.Journal of intellectual disability research : JIDR · 2012
    Article
13. Alkaptonuria: leading to the treasure in exceptions.JIMD reports · 2012
    Article

Corrections and comments

• Comment on
  Efficacy and safety of sirolimus in lymphangioleiomyomatosis.2011

Authors and funding

2 authors.

Funding

No grant is acknowledged in the PubMed record.

Abstract

PubMed holds no abstract for this paper.

Indexed as