Gene Therapy Led to “Robust Microdystrophin Expression” in Duchenne Muscular Dystrophy Patient
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Gene Therapy Led to “Robust Microdystrophin Expression” in Duchenne Muscular Dystrophy Patient

Duchenne muscular dystrophy (DMD) is an inherited disease that weakens a child’s muscles and eventually spreads throughout the child’s body. DMD is caused by mutations in the gene that helps…

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PGN-EDO51 Granted Orphan Drug Designation for Duchenne Muscular Dystrophy (DMD)
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PGN-EDO51 Granted Orphan Drug Designation for Duchenne Muscular Dystrophy (DMD)

Editor's Note: Chronic conditions and rare diseases don't discriminate, Patient Worthy and its partners are interested in amplifying the voices of those from all identities and backgrounds. If you have…

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CureDuchenne’s Cares Workshop: Atlanta, GA

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CureDuchenne Cares Workshop February 10, 2024 Atlanta, GA A FULL DAY EVENT FOCUSED ON IMPROVING THE QUALITY OF LIFE FOR INDIVIDUALS WITH DUCHENNE AND BECKER. CureDuchenne Cares is an interactive…

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Sarepta is Pushing to Expand Duchenne Muscular Dystrophy Drug Label Despite Missed Endpoints
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Sarepta is Pushing to Expand Duchenne Muscular Dystrophy Drug Label Despite Missed Endpoints

Sarepta Therapeutics’ treatment Elevidys (delandistrogene moxeparvovec-rokl) received conditional approval from the FDA in June 2023. The designation implies that if the drug is used in accordance with its label, it…

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Knowledge is Power: CureDuchenne Cares Educational Events Aim to Equip Families and Caregivers in the Becker and Duchenne Muscular Dystrophy Communities

CureDuchenne, a global leader in research and patient care for Duchenne muscular dystrophy (DMD), has been working to find and fund a cure for DMD for more than two decades.…

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SGT-003 Granted Orphan Drug Designation for Duchenne Muscular Dystrophy (DMD)

The research landscape for Duchenne muscular dystrophy (DMD) has been steadily increasing. Researchers are exploring more therapies, including gene therapies, that could transform the lives of those with this rare…

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Vamorolone Has Been FDA Approved to Treat Duchenne Muscular Dystrophy
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Vamorolone Has Been FDA Approved to Treat Duchenne Muscular Dystrophy

Vamorolone, now marketed as AGAMREE®, is termed a dissociative steroid therapy, as it has proven its potential to retain the muscle-strengthening and anti-inflammatory benefits of corticosteroids. On the other side…

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Rare Community Profiles: Destroy Duchenne: How Elijah Stacy is Working to Improve the Lives of People with Duchenne Muscular Dystrophy
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Rare Community Profiles: Destroy Duchenne: How Elijah Stacy is Working to Improve the Lives of People with Duchenne Muscular Dystrophy

Rare Community Profiles     Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families,…

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Death of Muscular Dystrophy Patient Potentially Caused by Adeno-Associated Virus That Delivered Gene Therapy
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Death of Muscular Dystrophy Patient Potentially Caused by Adeno-Associated Virus That Delivered Gene Therapy

For over four decades, the aspiration of gene therapy has been to develop novel therapies with the potential to enhance human health. Recombinant AAV (rAAV), a type of gene therapy,…

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CureDuchenne Launches the CureDuchenne Caregiver Course to Support Caregivers of People with DMD

  Since its founding 20 years ago, CureDuchenne has been tirelessly working to find and fund a cure for Duchenne muscular dystrophy (DMD). They have done this through supporting families,…

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FDA Clears Clinical Study to Evaluate BB-301 for Oculopharyngeal Muscular Dystrophy
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FDA Clears Clinical Study to Evaluate BB-301 for Oculopharyngeal Muscular Dystrophy

  Prior to launching a clinical trial, the FDA must clear an Investigational New Drug (IND) application. This allows the drug to be distributed across state lines, which is important…

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Two Affected with Congenital Muscular Dystrophy Strive for Rare Inclusion
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Two Affected with Congenital Muscular Dystrophy Strive for Rare Inclusion

  Congenital muscular dystrophy (CMD) affected individuals, Kelly Berger and Avery Roberts are making their voices heard. Together they hope to tackle misconceptions surrounding disability and give a loud voice…

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Rare Community Profiles: A Partnership Between CureDuchenne and PicnicHealth Bolsters the Power of CureDuchenne Link
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Rare Community Profiles: A Partnership Between CureDuchenne and PicnicHealth Bolsters the Power of CureDuchenne Link

Rare Community Profiles     Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…

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MP1032 Earns Orphan Drug Designation for DMD
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MP1032 Earns Orphan Drug Designation for DMD

As reported in Yahoo! Finance, the United States’ Food and Drug Administration (FDA) recently granted Orphan Drug designation to MP1032. This therapy, developed by clinical-stage biotech company MetrioPharm, is being…

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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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