Experimental Duchenne Muscular Dystrophy Treatment Earns Fast Track Designation
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Experimental Duchenne Muscular Dystrophy Treatment Earns Fast Track Designation

According to a story from PR Newswire, the biopharmaceutical company Avidity Biosciences, Inc., recently announced that its investigational therapy AOC 1044 has received the US Food and Drug Administration's Fast…

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MDA Clinical & Scientific Conference: Givinostat Improves Physical Function and Mobility in DMD
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MDA Clinical & Scientific Conference: Givinostat Improves Physical Function and Mobility in DMD

The Muscular Dystrophy Association (MDA) recently held its MDA Clinical & Scientific Conference in March 2023. During the conference, stakeholders in the community discussed research trends and clinical practices associated…

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How PepGen’s Jane Larkindale and Alayna Tress Advocate for Patient-Centricity in Rare Disease Drug Development
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How PepGen’s Jane Larkindale and Alayna Tress Advocate for Patient-Centricity in Rare Disease Drug Development

Contributed by Jane Larkindale and Alayna Tress While millions of people globally are living with a rare disease, patients often find it difficult to feel seen or heard throughout their…

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In Utero DMD Therapy Was Unsuccessful in Murine Models
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In Utero DMD Therapy Was Unsuccessful in Murine Models

The current standards-of-care for those with Duchenne muscular dystrophy (DMD) include corticosteroids, immunosuppressive treatments, physical therapy, and occupational therapy (among others). Gene therapy has the potential to change the treatment…

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CureDuchenne Has Launched a Certified Occupational Therapist Program: An Interview with Jennifer Wallace Valdes, PT (Pt. 2)
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CureDuchenne Has Launched a Certified Occupational Therapist Program: An Interview with Jennifer Wallace Valdes, PT (Pt. 2)

Before you read on, don't forget to check out Part 1 of our interview with Jennifer Wallace Valdes, PT. In Part 1, Jennifer discusses her background, what Duchenne muscular dystrophy (DMD) is,…

Continue Reading CureDuchenne Has Launched a Certified Occupational Therapist Program: An Interview with Jennifer Wallace Valdes, PT (Pt. 2)
CureDuchenne Has Launched a Certified Occupational Therapist Program: An Interview with Jennifer Wallace Valdes, PT (Pt. 1)
Photo courtesy of CureDuchenne

CureDuchenne Has Launched a Certified Occupational Therapist Program: An Interview with Jennifer Wallace Valdes, PT (Pt. 1)

Occupational therapy for individuals with Duchenne muscular dystrophy (DMD) requires a unique and specialized approach to maximize benefit and reduce harm. It is incredibly important that those performing occupational therapy…

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CureDuchenne Starts New Occupational Therapist Certification Program to Enhance Duchenne Muscular Dystrophy Treatment
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CureDuchenne Starts New Occupational Therapist Certification Program to Enhance Duchenne Muscular Dystrophy Treatment

According to a story from Patient Worthy partner CureDuchenne, the organization has recently launched its CureDuchenne Occupational Therapist Certification Program. This program is intended to give occupational therapists advanced skills…

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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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