Rare Diseases [NEW TOPIC PAGE]

Main|Search|PHGKB



From National Institutes of Health Web Sites

---

• Accelerating evidence gathering and approval of precision medicine therapies: the FDA takes aim at rare mutations
  AM Issa et al, Genetics in Medicine, July 10, 2018
• Clinical Trials Bring Hope to Kids with Spinal Muscular Atrophy
  F Collins, NIH Director's blog, Nov 21, 2017
• February is American Heart Month. What are rare diseases involving the heart. 
  NIH Office of Rare Diseases
• Molecular Answers Found for a Mysterious Rare Immune Disorder
  Francis Collins, NIH Director, Blog Post, March 29, 2016
• Notice of Intent to Publish a Funding Opportunity Announcement for Rare Disease Cohorts in Heart, Lung, Blood and Sleep Disorders
  NHLBI, Apr 2018
• Rare Disease 2019 Information Packet
  NIH, October 2018
• Rare Disease Day at NIH
  On February 27, 2015, the National Institutes of Health (NIH) will celebrate the eighth annual Rare Disease Day with a day-long celebration
• Rare Disease Day, February 27, 2017 
  NIH, National Centers for Advancement of Translational Sciences
• Research Round-up: Rare Disease Research
  Researchers seek to expand our understanding and develop new treatments, NICHD< February 1, 2016