Muscular dystrophy

Last Posted: Dec-02-2016 8AM

 Muscular Dystrophy expanded expanded

Muscling out gene mutations
KA WHitehead, Sci Trans Medicine, November 30, 2016

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

Economic Evaluation in Duchenne Muscular Dystrophy: Model Frameworks for Cost-Effectiveness Analysis.
Landfeldt Erik et al. PharmacoEconomics 2016 Oct

More information on this topic

Last Updated: Nov 30, 2016

• 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
  AS Kesselheim et al, JAMA October 25, 2016
• FDA commissioner calls for Sarepta drug study to be retracted
  StatNews, September 22, 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
• Advances in gene therapy for muscular dystrophies.
  Abdul-Razak Hayder et al. F1000Research 2016
• Current and emerging treatment strategies for Duchenne muscular dystrophy.
  Mah Jean K et al. Neuropsychiatric disease and treatment 2016 1795-807
• FDA approves Sarepta’s controversial drug for Duchenne muscular dystrophy
  E Silverman, Stat News, September 19, 2016
• The emerging role of viral vectors as vehicles for DMD gene editing
  I Maggio et al, May 23, 2016
• Clinical Follow-Up For Duchenne Muscular Dystrophy Newborn Screening: A Proposal.
  Kwon Jennifer M et al. Muscle & nerve 2016 May
• Green light for Duchenne muscular dystrophy drug
  BBC News, April 15, 2016
• A systematic review of risk factors associated with muscular dystrophies.
  Barakat-Haddad Caroline et al. Neurotoxicology 2016 Mar
• Clinical utility of serum biomarkers in Duchenne muscular dystrophy.
  Hathout Yetrib et al. Clinical proteomics 2016 9

• CDC Information (4)
• CDC-Authored Pub (5)

• Human (14)
• Pathogen (0)

• Human (141)
• Pathogen (0)

• Human (22)
• Pathogen (0)

• Guidelines (3)
• Tier Table (0)
• Synthesis (2)

• Huamn (1)
• Pathogen (0)

Relevant Resources

• Genetics and Genomics Competencies Center (G2C2)
• NIH Genetic Testing Registry
• NIH MedGen Database
• Online Mendelian Inheritance in Man (OMIM)
• PharmGKB