Editor’s Choice: A New CRISPR Trial, Revelations About Zika-Related GBS, and Parents Taking Action

Happy Thursday! This week, we have details on the beginnings of a new trial testing CRISPR gene editing for Duchenne muscular dystrophy, how parents can play a critical role in…

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The FDA Grants Approval to Begin Dosing First-in-Human CRISPR Technology to Treat Duchenne Muscular Dystrophy
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The FDA Grants Approval to Begin Dosing First-in-Human CRISPR Technology to Treat Duchenne Muscular Dystrophy

   Business Wire published an August 10th news release announcing that Cure Rare Disease, a non-profit based in Boston, Massachusetts received FDA approval to administer CRD-TMH-001 (CRD), its first-ever therapeutic.…

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Rare Disease Patients Still Waiting for Crowdfunding Plan in India
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Rare Disease Patients Still Waiting for Crowdfunding Plan in India

As we know, many treatments for rare diseases are quite expensive - and this price doesn't even factor in travel, lodging, appointments, and more. To help with this, the government…

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Making a Difference: An Interview With Debra Miller on the Founding of CureDuchenne
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Making a Difference: An Interview With Debra Miller on the Founding of CureDuchenne

CureDuchenne is one of Patient Worthy's partner organizations. Patient Worthy partners with a variety of rare disease and patient-oriented non-profits in order to collaborate and help promote one another's activities.…

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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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