Last Posted: Oct 04, 2018
- A new era of patient focused innovation
NORD, Rare Diseases summit, October 15-16,2018 - The Effect of a Pediatric Rare Disease on Subscriber Retention Rates for Commercial Health Insurers in the United States.
Kuester Melanie K et al. Journal of managed care & specialty pharmacy 2018 Sep 1-9 - Wellcome Sanger Institute at 25: how the genomic revolution is changing medicine
Wellcome Sanger, September 2018 - [Family Therapeutic Trajectories: rare hereditary diseases involving long-term suffering].
Aureliano Waleska de Araújo et al. Ciencia & saude coletiva 2018 Feb 23(2) 369-380 - Integrated genetic and pharmacologic interrogation of rare cancers.
Hong Andrew L et al. Nature communications 2016 711987 - International working group identifies need for newborn screening for mucopolysaccharidosis type I but states that existing hurdles must be overcome.
Parini Rossella et al. Acta paediatrica (Oslo, Norway : 1992) 2018 Sep - Solving the genome puzzle: With advances in gene technology helping to diagnose very rare diseases, has the new era of personalised medicine finally arrived?
L Geddes, The Guardian, September 21, 2018 - The NIH Undiagnosed Diseases Network expands
NIH News, September 234, 2018 - The Role of Gene Therapy in the Treatment of Retinal Diseases: A Review.
Campa C et al. Current gene therapy 2017 17(3) 194-213 - What Precision Medicine Can Learn from Rare Genetic Disease Research and Translation.
Tabor Holly K et al. AMA journal of ethics 2018 Sep 20(9) E834-840
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