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What are the new drugs for Duchenne muscular dystrophy?

Medically reviewed by Drugs.com. Last updated on Sep 3, 2020.

Official Answer

by Drugs.com

The new drugs for the treatment of Duchenne muscular dystrophy are Viltepso, Vyondys 53, Exondys 51, and Emflaza.

Duchenne muscular dystrophy (DMD) is a genetic disease that causes progressive weakness and loss of skeletal and heart muscles. DMD is caused by mutations in the DMD gene.

The medicines below have been FDA approved for the treatment of Duchenne muscular dystrophy:

Viltepso (viltolarsen) Injection

  • FDA Approved: August 12, 2020
  • Company: NS Pharma, Inc.

Viltepso is an antisense oliogonucleotide indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping.

Vyondys 53 (golodirsen) Injection

  • FDA Approved: December 12, 2019
  • Company: Sarepta Therapeutics

Vyondys 53 is an antisense oligonucleotide indicated for the treatment of DMD in patients with a confirmed mutation of the DMD gene amenable to exon 53 skipping.

Emflaza (deflazacort) Tablets and Oral Suspension

  • FDA Approved: February 9, 2017
  • Company: PTC Therapeutics, Inc.

Emflaza is a glucocorticoid indicated for the treatment of DMD in patients 2 years of age and older.

Exondys 51 (eteplirsen) Injection

  • FDA Approved: September 19, 2016
  • Company: Sarepta Therapeutics

Exondys 51 is an antisense oligonucleotide indicated for the treatment of DMD in patients with a confirmed mutation of the DMD gene amenable to exon 51 skipping.

Prednisone is a glucocorticoid that is also used in the treatment of DMD.

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