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  • Updated 03.27.2018
  • Released 03.27.2018
  • Expires For CME 03.27.2021

Neuromuscular diseases: high cost of new treatments


This article includes discussion of the high cost of new treatments for neuromuscular diseases and the Orphan Drug Act.


Neuromuscular diseases are on the forefront of therapeutic development. Three new disease-modifying therapies have been approved in the past 12 months for rare neuromuscular disorders, an unprecedented pace for any area of neurology. The favorable regulatory landscape for orphan disease has provided the impetus to invest in these disorders. However, the cost and intensity of these therapies have provided new challenges to physicians treating these debilitating illnesses. In this article, the authors describe the challenges and identify some solutions moving forward.

Key points

• Incentives within the Orphan Drug Act have promoted research in rare neuromuscular diseases.

• New disease-modifying therapies in neuromuscular disease are difficult to implement given the cost and route of administration.

• Initial implementation in multidisciplinary clinics may be the most feasible approach.

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