Duchenne Muscular Dystrophy

• FDA approval · 2020-07-10
  FDA Approved: EMFLAZA — Supplemental Application (PTC Therapeutics, Inc.)

  FDA approved a supplemental application for EMFLAZA (deflazacort) from PTC Therapeutics, expanding/confirming its labeling for Duchenne musc…

• FDA approval · 2020-07-07
  FDA Approved: EXONDYS 51 — Supplemental Application (Sarepta Therapeutics, Inc.)

  The FDA approved a supplemental application for Exondys 51 (eteplirsen) from Sarepta Therapeutics, Inc.

• FDA approval · 2019-12-12
  FDA Approved: VYONDYS 53 — Original Application (Sarepta Therapeutics, Inc.)

  The FDA approved Sarepta Therapeutics' golodirsen (VYONDYS 53) as the original application for treatment of Duchenne muscular dystrophy due …

• FDA approval · 2019-06-07
  FDA Approved: EMFLAZA — Supplemental Application (PTC Therapeutics, Inc.)

  The FDA approved a supplemental application for EMFLAZA (deflazacort) submitted by PTC Therapeutics, Inc.

• FDA approval · 2018-10-11
  FDA Approved: EXONDYS 51 — Supplemental Application (Sarepta Therapeutics, Inc.)

  The FDA approved Sarepta Therapeutics' supplemental application for Exondys 51 (eteplirsen) for Duchenne muscular dystrophy.

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• CLINICAL_TRIALS approval · 2018-02-28
  Clinical Trial Update: Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy

  PTC Therapeutics' deflazacort (Emflaza) Expanded Access Program for Duchenne Muscular Dystrophy has been approved for marketing.

• FDA approval · 2018-02-08
  FDA Approved: EXONDYS 51 — Supplemental Application (Sarepta Therapeutics, Inc.)

  The FDA approved a supplemental application for Exondys 51 (eteplirsen) submitted by Sarepta Therapeutics, Inc.

• FDA approval · 2017-02-22
  FDA Approved: EXONDYS 51 — Supplemental Application (Sarepta Therapeutics, Inc.)

  FDA approves a supplemental application for Exondys 51 (eteplirsen) from Sarepta Therapeutics.

• FDA approval · 2017-02-09
  FDA Approved: EMFLAZA — Original Application (PTC Therapeutics, Inc.)

  The FDA approved EMFLAZA (deflazacort) from PTC Therapeutics via original NDA for the treatment of Duchenne muscular dystrophy in patients a…

• FDA approval · 2017-02-09
  FDA Approved: EMFLAZA — Original Application (PTC Therapeutics, Inc.)

  FDA approved EMFLAZA (deflazacort) in an original application from PTC Therapeutics for Duchenne muscular dystrophy.

• FDA approval · 2016-09-19
  FDA Approved: EXONDYS 51 — Original Application (Sarepta Therapeutics, Inc.)

  FDA approved Exondys 51 (eteplirsen) from Sarepta Therapeutics as the original NDA for Duchenne muscular dystrophy amenable to exon 51 skipp…

• EMA approval · 2014-05-23
  European Medicines Agency recommends first-in-class medicine for treatment of Duchenne muscular dystrophy

  EMA recommends a first-in-class medicine for Duchenne muscular dystrophy, signaling potential approval for a novel mechanism in this indicat…