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The Pathway to New Therapies: Duchenne Muscular Dystrophy

 (17 Pages)

In a special report by Liftstream we look at one of the most active areas in rare disease research, Duchenne Muscular Dystrophy (DMD).  DMD and its milder form, Becker Muscular Dystrophy (BMD), are genetic disorders affecting approximately one in 3500 new born males. There are currently estimated to be 300,000 people worldwide who currently live with Duchenne. At present there is no cure and treatment is mainly palliative. Liftstream has been able to gain comment and opinion from thought leaders from both patient advocacy groups and the biopharmaceutical industry to develop this extensive report.

The report outlines major hurdles for new clinically proven therapies, including the regulatory approval process, manufacturing challenges, pricing and reimbursement, and market access.

  • The patient perspective
  • Novel therapeutic approaches for DMD
  • Exon skipping
  • Utrophin modulation
  • After clinical trials
  • Access to medicine
  • Market access stakeholders
  • Manufacturing challenges in rare diseases
  • Patient registries
  • Aligning stakeholders in rare diseases

An appendix of all images in the report is available on request.  


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