Patient organizations and research on rare diseases

N Engl J Med. 2011 Apr 28;364(17):1670-1. doi: 10.1056/NEJMe1102290. Epub 2011 Mar 16.
No abstract available

Publication types

  • Comment
  • Editorial

MeSH terms

  • Female
  • Foundations
  • Humans
  • Lymphangioleiomyomatosis / drug therapy*
  • Rare Diseases*
  • Self-Help Groups*
  • Sirolimus / therapeutic use*
  • TOR Serine-Threonine Kinases / antagonists & inhibitors

Substances

  • MTOR protein, human
  • TOR Serine-Threonine Kinases
  • Sirolimus