A Parent’s Take on Marriage While Having a Child with Duchenne Muscular Dystrophy
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A Parent’s Take on Marriage While Having a Child with Duchenne Muscular Dystrophy

  Betty Vertin recently wrote an article discussing her experience as a parent of a child with Duchenne muscular dystrophy. She shares the lessons she has learned in order to…

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Study Finds Blocking Sphingolipids Counteracts Muscular Dystrophy in Mice Models
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Study Finds Blocking Sphingolipids Counteracts Muscular Dystrophy in Mice Models

According to a recent article, a research team discovered that the inhibition of sphingolipid synthesis on mice models of Duchenne muscular dystrophy can counteract the certain symptoms of the disease.…

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Clinical Trial of Vamorolone Receives Orphan Grant Funding
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Clinical Trial of Vamorolone Receives Orphan Grant Funding

Funding for clinical trials can be an issue, especially for rare disease studies. Luckily, there are programs that exist to help with the financials, such as the "Clinical Studies of…

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September 7 is World Duchenne Awareness Day: Spreading Duchenne Muscular Dystrophy Awareness
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September 7 is World Duchenne Awareness Day: Spreading Duchenne Muscular Dystrophy Awareness

September 7, 2021 will be recognized as World Duchenne Awareness Day. This day will be set aside in recognition of awareness surrounding Duchenne muscular dystrophy, a rare and debilitating genetic…

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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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