Webinar: Using the Rare Disease Cures Accelerator for a DMD Progression Model
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Webinar: Using the Rare Disease Cures Accelerator for a DMD Progression Model

On July 21, 2021, Patient Worthy attended an online webinar presentation titled "How RDCA-DAP Can Help Inform Optimal Trial Design in Progressive Rare Disease." Organized by the Critical Path Institute…

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The First Patient in China to be Accepted Under its 2019 Compassionate Use Policy

Beijing, China: According to an article in a sister publication of Sixth Tone, a woman living in China who has been treated during the past fourteen years for paroxysmal nocturnal…

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Experimental Duchenne Muscular Dystrophy Treatment Earns Rare Pediatric Disease Designation
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Experimental Duchenne Muscular Dystrophy Treatment Earns Rare Pediatric Disease Designation

According to a story from apnews.com, the biopharmaceutical company FibroGen, Inc., has recently announced that its investigational treatment pamrevlumab has earned Rare Pediatric Disease designation from the US Food and…

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Investigative Therapy for Duchenne Muscular Dystrophy is in a Phase 3 Clinical Trial
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Investigative Therapy for Duchenne Muscular Dystrophy is in a Phase 3 Clinical Trial

FibroGen has just announced that their investigative therapy pamrevlumab, for Duchenne muscular dystrophy (DMD), has received Rare Pediatric Disease designation from the FDA. The treatment already was given Fast Track…

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SGT-001 Safety and Efficacy Data Available from IGNITE DMD Clinical Trial
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SGT-001 Safety and Efficacy Data Available from IGNITE DMD Clinical Trial

Since its inception, life sciences company Solid Biosciences Inc. has been working to develop treatment options for patients with Duchenne muscular dystrophy (DMD). According to a recent press release, the…

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This Phase 3 Study Will Evaluate Pamrevlumab as a Treatment for DMD
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This Phase 3 Study Will Evaluate Pamrevlumab as a Treatment for DMD

Pamrevlumab is a first-in-class antibody that treats Duchenne muscular dystrophy (DMD) by inhibiting the effects and activity of connective tissue disorder growth factor (CTGF). It is being developed by FibroGen,…

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Updates Available on Givinostat Clinical Development for DMD

During the virtual XVIII International Conference on Duchenne and Becker Muscular Dystrophy, specialty pharmaceutical company Italfarmaco Group ("Italfarmaco") shared updates regarding various Givinostat clinical development programs. The treatment is designed…

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The First Duchenne Muscular Dystrophy Patient Receives Gene Therapy Treatment in Pfizer’s Phase 3 Trial

The very first patient has just been dosed in Pfizer's Phase 3 clinical trial for Duchenne muscular dystrophy (DMD). The trial is called CIFFREO. This patient received the therapy at a…

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Avidity Announces 2021 Pipeline Updates For DM1, FSHD, DMD
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Avidity Announces 2021 Pipeline Updates For DM1, FSHD, DMD

In early January 2021, biopharmaceutical company Avidity Biosciences ("Avidity") announced updates from its 2021 pipeline. Currently, Avidity is working to create a line of Antibody Oligonucleotide Conjugates (AOCs), therapeutic options…

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