Neuromuscular diseases: high cost of new treatments

Nicholas E Johnson MD (

Dr. Johnson of the University of Utah received consulting fees from AMO Pharma, University of Utah, and AveXis and a research grant from Biogen Idec.

Amber K Stock MPH (

Ms. Stock of the American Academy of Neurology has no relevant financial relationships to disclose.

Amy Kaloides (

Ms. Kaloides of the American Academy of Neurology has no relevant financial relationships to disclose.

Emma Ciafaloni MD, editor. (

Dr. Ciafaloni of the University of Rochester received personal compensation for serving on advisory boards and/or as a consultant for Avexis, Biogen, Pfizer, PTC Therapeutics, Sarepta, Ra pharma, Wave, and Strongbridge Biopharma; and for serving on a speaker’s bureau for Biogen. Dr Ciafaloni also received research and/or grant support from Orphazyme, PTC Therapeutics, Santhera, and Sarepta.

Originally released March 27, 2018; expires March 27, 2021

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.

The content you are trying to view is available only to logged in, current MedLink Neurology subscribers.

If you are a subscriber, please log in.

If you are a former subscriber or have registered before, please log in first and then click select a Service Plan or contact Subscriber Services. Site license users, click the Site License Acces link on the Homepage at an authorized computer.

If you have never registered before, click Learn More about MedLink Neurology  or view available Service Plans.

Find out how you can join MedLink Neurology