Home | Neuromuscular | Duchenne muscular dystrophy (DMD) | Congress activities | American Academy of Neurology Annual Meeting (AAN) | AAN 2023 oral presentation: Evaluation of the biodistribution, efficacy, and side-effect profile of deflazacort, prednisolone and vamorolone in a DMD mouse model

AAN 2023 oral presentation: Evaluation of the biodistribution, efficacy, and side-effect profile of deflazacort, prednisolone and vamorolone in a DMD mouse model

This oral presentation at AAN 2023 explored the use of 3 corticosteroids in a Duchenne muscular dystrophy (DMD) mouse model. The study monitored pharmacokinetics and biodistribution, the efficacy on muscle function and damage, and explored potential side effects

  • Evaluate the differences between 3 corticosteroids in a DMD mouse model
  • Explore how blood/brain biodistribution differs between these corticosteroids
  • Discover biological efficacy of the corticosteroids, including how they impact blood count, muscle function and muscle damage
  • Compare the safety profile of each corticosteroid

Deflazacort is a corticosteroid indicated for the treatment of DMD in patients 2 years of age and older. PTC Therapeutics is the Marketing Authorization Holder for deflazacort in the US only. Registration conditions differ internationally, and prescribing information may vary depending on local approval in each country. The US Prescribing Information for deflazacort is available here. Please refer to your local country guidance for more information.

For medical information, product complaints, or to report an adverse event, please call 1-866-562-4620 or email at usmedinfo@ptcbio.com. You may also report adverse events directly to FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

This oral presentation was developed and funded by PTC Therapeutics for AAN 2023.

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MED-ALL-DMD-2300168 | September 2023
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