Dystrophie musculaire de Duchenne Dystrophie musculaire de Duchenne Dystrophie musculaire de Duchenne

Treatments

EMBARK, gene therapy for the treatment of DMD
By Marie
In News, Research, Treatments
Posted 4 October 2021

EMBARK, gene therapy for the treatment of DMD

Sarepta Therapeutics announces initiation of EMBARK, a global p0ivotal study of SRP-9001, gene therapy for the treatment of DMD (Duchenne Muscular Dystrophy) Original press release > [...]

Part B of MOMENTUM study of SRP-5051 in patients with DMD
By Marie
In Research, Treatments
Posted 27 September 2021

Part B of MOMENTUM study of SRP-5051 in patients with DMD

Sarepta Therapeutics to initiate Part B of MOMENTUM study of SRP-5051 in patients with Duchenne muscular dystrophy (DMD) amenable to Exon 51 skipping following positive interactions with FDA [...]

ENDEAVOR, gene therapy for DMD
By Marie
In News, Research, Treatments
Posted 20 May 2021

ENDEAVOR, gene therapy for DMD

Sarepta Therapeutics’ investigational gene therapy for the treatment of Duchenne muscular dystrophy, SRP-9001, demonstrates robust expression and consistent safety profile using Sarepta’s [...]

Positive clinical results from phase 2 MOMENTUM study
By Marie
In News, Research, Treatments
Posted 4 May 2021

Positive clinical results from phase 2 MOMENTUM study

Sarepta Therapeutics reports positive clinical results from phase 2 MOMENTUM study of SRP-5051 in patients with Duchenne muscular dystrophy amenable to skipping exon 51 Results suggest a highly [...]

News from Vamorolone in Duchenne Muscular Dystrophy
By Marie
In Research, Treatments
Posted 3 March 2021

News from Vamorolone in Duchenne Muscular Dystrophy

Santhera Announces Completion of First 6-Month Period of Pivotal VISION-DMD Trial with Vamorolone in Duchenne Muscular Dystrophy Original Press release > Pratteln, Switzerland, March 3, 2021 [...]

FDA Approval of AMONDYS 45 (casimersen)
By Marie
In News, Treatments
Posted 25 February 2021

FDA Approval of AMONDYS 45 (casimersen)

Sarepta Therapeutics announces FDA approval of AMONDYS 45 (casimersen) injection to treat Duchenne muscular dystrophy (DMD) in patients amenable to skipping exon 45. Feb. 25, 2021, >Original [...]

Italfarmaco provides an update on Givinostat
By Marie
In Research, Treatments
Posted 22 February 2021

Italfarmaco provides an update on Givinostat

Italfarmaco provides an update on Givinostat, an ongoing clinical program, in an oral presentation at XVIII International Conference on Duchenne and Becker Muscular Dystrophy. Feb. 22, 2021, [...]

Sarepta announces top-line results for its investigational gene therapy
By Marie
In in the Medias, Research, Treatments
Posted 8 January 2021

Sarepta announces top-line results for its investigational gene therapy

Sarepta Therapeutics announces top-line results for part 1 of study 102 evaluating SRP-9001, its investigational gene therapy for the treatment of Duchenne muscular dystrophy (DMD)   Jan. 7, [...]

Pfizer doses the first participant with Its gene therapy
By Marie
In News, Research, Treatments
Posted 7 January 2021

Pfizer doses the first participant with Its gene therapy

Pfizer doses the first participant in phase 3 study for Duchenne muscular dystrophy (DMD) investigational gene therapy. January 07, 2021, > Original press release < Pfizer Inc. announced [...]

Positive clinical results from MOMENTUM
By Marie
In Treatments
Posted 7 December 2020

Positive clinical results from MOMENTUM

Sarepta Therapeutics announces positive clinical results from MOMENTUM, a phase 2 clinical trial of SRP-5051 in patients with Duchenne muscular dystrophy amenable to skipping exon 51. Original [...]

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Articles récents

  • The global day of giving
  • EMBARK, gene therapy for the treatment of DMD
  • Part B of MOMENTUM study of SRP-5051 in patients with DMD
  • World Duchenne Awareness Day 2021
  • ENDEAVOR, gene therapy for DMD

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