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Sarepta announces top-line results for its investigational gene therapy

SRP-9001 gene therapy DMD

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, 2021, >Original […]

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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 that the […]

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 press release > […]


The power of giving back

GivingTuesday December 1rst

Giving Tuesday – the global day of giving Join us for the 8th annual Giving Tuesday on December 1rst, 2020! GivingTuesday is a global day of giving that happens each […]

The development of edasalonexent will be stopped

development edasalonexent stopped

Catabasis Pharmaceuticals announces top-line results for the phase 3 PolarisDMD trial of edasalonexent in Duchenne muscular dystrophy.   PolarisDMD Trial Did Not Achieve Primary or Secondary Endpoints – The development […]

Santhera to discontinue Phase 3 SIDEROS study

discontinue Phase 3 SIDEROS

Santhera to discontinue Phase 3 SIDEROS study and development of Puldysa® in Duchenne Muscular Dystrophy (DMD) and focus on vamorolone. Original Press Release > Pratteln, Switzerland, October 6, 2020 Santhera […]