Muscular Dystrophy || Public Health Genomics Knowledge Base (v1.2)

Public Health Genomics Knowledge Base (v1.2)




Last Posted: Feb 10, 2017

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• 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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