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First Drug for Duchenne Muscular Dystrophy Gets 'Conditional Approval' in Europe

Submitted by jcook on Mon, 05/02/2016 - 10:50

CHICAGO, Aug. 4, 2014 — A drug called ataluren (brand name Translarna), in development to treat a subset of patients with Duchenne muscular dystrophy (DMD) has received "conditional approval" in the European Union.

This is the first drug specifically developed for and tested in Duchenne muscular dystrophy to receive approval in any country. PTC Therapeutics, the drug’s developer, announced the approval in an Aug. 4, 2014, press release.
PTC Therapeutics Receives Conditional Approval in the European Union for Translarna™ for the treatment of Nonsense Mutation Duchenne Muscular Dystrophy

Submitted by jcook on Mon, 05/02/2016 - 10:51

- The First Treatment Approved for DMD -

SOUTH PLAINFIELD, N.J., Aug. 4, 2014 — PTC Therapeutics, Inc. today announced that the European Commission has granted conditional marketing authorization for Translarna™ (ataluren), in the European Union (EU) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients aged five years and older.
International Consortium Provides $2M Funding for Phase 1 Clinical Trial of ReveraGen DMD Drug

Submitted by jcook on Mon, 05/02/2016 - 11:16

SILVER SPRING, Maryland; CHICAGO, LONDON, United Kingdom, July 17, 2014 -- Four nonprofit organizations, the Muscular Dystrophy Association (USA), Joining Jack Foundation (UK), The Duchenne Research Fund (UK) and the Duchenne Children’s Trust (UK) have joined together to provide Phase 1 clinical trial funding for the first-in-human studies of VBP15, the lead compound of ReveraGen BioPharma for Duchenne muscular dystrophy.
Sarepta Therapeutics Reports Long-Term Outcomes through 144 Weeks from Phase IIb Study of Eteplirsen in Duchenne Muscular Dystrophy

Submitted by jcook on Mon, 05/02/2016 - 10:52

CAMBRIDGE, Mass. – July 10, 2014 – Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a developer of innovative RNA-based therapeutics, today announced data through Week 144 from Study 202, a Phase IIb open-label extension study of eteplirsen in patients with Duchenne muscular dystrophy (DMD). After nearly three years of follow up, results on the 6-minute walk test (6MWT) showed a decline in walking ability at a rate slower than would be expected based on available DMD natural history data.
Isis Pharmaceuticals Initiates Phase 1 Study of ISIS-DMPK Rx to Treat Myotonic Dystrophy Type 1

Submitted by jcook on Mon, 05/02/2016 - 11:17

Isis Earns $14 Million Milestone Payment from Biogen Idec ISIS-DMPKRx is the Second Generation 2.5 Antisense Drug to Enter the Clinic
ALS: Experimental Drug Tirasemtiv Fails To Show Efficacy in Phase 2b Trial

Submitted by jcook on Mon, 05/02/2016 - 11:18

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Press Releases

First Drug for Duchenne Muscular Dystrophy Gets 'Conditional Approval' in Europe

PTC Therapeutics Receives Conditional Approval in the European Union for Translarna™ for the treatment of Nonsense Mutation Duchenne Muscular Dystrophy

- The First Treatment Approved for DMD -

International Consortium Provides $2M Funding for Phase 1 Clinical Trial of ReveraGen DMD Drug

Sarepta Therapeutics Reports Long-Term Outcomes through 144 Weeks from Phase IIb Study of Eteplirsen in Duchenne Muscular Dystrophy

Isis Pharmaceuticals Initiates Phase 1 Study of ISIS-DMPK Rx to Treat Myotonic Dystrophy Type 1

ALS: Experimental Drug Tirasemtiv Fails To Show Efficacy in Phase 2b Trial

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About Us

Press Releases

First Drug for Duchenne Muscular Dystrophy Gets 'Conditional Approval' in Europe

PTC Therapeutics Receives Conditional Approval in the European Union for Translarna™ for the treatment of Nonsense Mutation Duchenne Muscular Dystrophy

- The First Treatment Approved for DMD -

International Consortium Provides $2M Funding for Phase 1 Clinical Trial of ReveraGen DMD Drug

Sarepta Therapeutics Reports Long-Term Outcomes through 144 Weeks from Phase IIb Study of Eteplirsen in Duchenne Muscular Dystrophy

Isis Pharmaceuticals Initiates Phase 1 Study of ISIS-DMPK Rx to Treat Myotonic Dystrophy Type 1

ALS: Experimental Drug Tirasemtiv Fails To Show Efficacy in Phase 2b Trial

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