Last Posted: Feb 10, 2017
- FDA approves drug to treat Duchenne muscular dystrophy
Reuters Health, February 9, 2017 - Researchers find genetic cause of new type of muscular dystrophy
Medical Xpress, February 9, 2017 - DMDtoolkit: a tool for visualizing the mutated dystrophin protein and predicting the clinical severity in DMD.
Zhou Jiapeng et al. BMC bioinformatics 2017 Feb 18(1) 87 - Using Administrative Data to Ascertain True Cases of Muscular Dystrophy: Rare Disease Surveillance.
Smith Michael G et al. JMIR public health and surveillance 2017 Jan 3(1) e2 - Breast cancer genetic counseling among Dutch patients from Turkish and Moroccan descent: participation determinants and perspectives of patients and healthcare professionals.
Baars J E et al. Journal of community genetics 2017 Jan - Muscling out gene mutations
KA WHitehead, Sci Trans Medicine, November 30, 2016 - Economic Evaluation in Duchenne Muscular Dystrophy: Model Frameworks for Cost-Effectiveness Analysis.
Landfeldt Erik et al. PharmacoEconomics 2016 Oct - Knowledge of carrier status and barriers to testing among mothers of sons with Duchenne or Becker muscular dystrophy.
Bogue Lauren et al. Neuromuscular disorders : NMD 2016 Sep - Approving a Problematic Muscular Dystrophy Drug: Implications for FDA Policy.
Kesselheim Aaron S et al. JAMA 2016 Oct - Approving a Problematic Muscular Dystrophy Drug- Implications for FDA Policy
AS Kesselheim et al, JAMA October 25, 2016 - FDA commissioner calls for Sarepta drug study to be retracted
StatNews, September 22, 2016 - Advances in gene therapy for muscular dystrophies.
Abdul-Razak Hayder et al. F1000Research 2016 5 - Current and emerging treatment strategies for Duchenne muscular dystrophy.
Mah Jean K et al. Neuropsychiatric disease and treatment 2016 121795-807 - FDA approves Sarepta’s controversial drug for Duchenne muscular dystrophy
E Silverman, Stat News, September 19, 2016 - For two brothers with Duchenne, an FDA drug approval brings joy and relief
J Delviscio, StatNews, September 20, 2016 - Sarepta to charge $300K for Duchenne drug. ‘We tried to be reasonable,’ CEO says
E Silverman, StatNews, September 19, 2016
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