duchenne muscular dystrophy

Momentum Builds Across Biotech Pipeline: Seizure Breakthrough, Cell Therapy Revival, and Obesity Medicine Gains

Momentum Builds Across Biotech Pipeline: Seizure Breakthrough, Cell Therapy Revival, and Obesity Medicine Gains

The biotechnology sector experienced significant positive developments this week, with Xenon Pharmaceuticals' seizure medication achieving a decisive late-stage victory while regulatory pathways accelerated for several other promising therapies. According to…

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Aucta Pharmaceuticals Launches PYQUVI, Expands Branded‑Generic Portfolio in Rare Disease Care

Aucta Pharmaceuticals Launches PYQUVI, Expands Branded‑Generic Portfolio in Rare Disease Care

As reported on the Manila Times, Aucta Pharmaceuticals has officially introduced PYQUVI™ (deflazacort) oral suspension 22.75 mg/mL, marking the company’s entry into the U.S. commercial branded‑generic market. The therapy, released…

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Biotech Innovation Accelerates: Four Companies Show Promising Pipeline Progress

Biotech Innovation Accelerates: Four Companies Show Promising Pipeline Progress

The biotech sector witnessed significant momentum as four clinical-stage companies announced compelling data across muscular dystrophy, immunology, and obesity treatment areas, triggering substantial stock market gains and investor enthusiasm for…

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Long-Term Study Shows Givinostat’s Promise for Duchenne Muscular Dystrophy Treatment

Long-Term Study Shows Givinostat’s Promise for Duchenne Muscular Dystrophy Treatment

ITF Therapeutics has announced the publication of encouraging long-term data supporting the efficacy and safety of givinostat as a treatment for Duchenne muscular dystrophy (DMD), according to a recent article…

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Brogidirsen and the Promise of Exon 44 Skipping: New Horizons in RNA Therapy for Duchenne Muscular Dystrophy

Brogidirsen and the Promise of Exon 44 Skipping: New Horizons in RNA Therapy for Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a devastating, inherited muscle-wasting disorder caused by mutations in the DMD gene, leading to an absence of functional dystrophin protein. While current standards of care,…

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Real-World Outcomes of Ataluren in Duchenne Muscular Dystrophy: Evidence from the STRIDE Registry

Real-World Outcomes of Ataluren in Duchenne Muscular Dystrophy: Evidence from the STRIDE Registry

Duchenne Muscular Dystrophy (DMD) is a severe, progressive neuromuscular disease that primarily affects boys, leading to muscle weakness, loss of ambulation, and premature death. Among therapeutic advances, ataluren has emerged…

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Achieving Global Excellence in Duchenne Muscular Dystrophy Care: The Accredited Duchenne Centers Program

Achieving Global Excellence in Duchenne Muscular Dystrophy Care: The Accredited Duchenne Centers Program

The Accredited Duchenne Centers (ADC) Program, spearheaded by the World Duchenne Organization (WDO), is a pioneering global initiative designed to standardize and elevate care for individuals living with Duchenne Muscular…

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Pfizer Acknowledges the Death of a Young Boy Who Died One Year After Participating in its Phase II Clinical Trial to Treat DMD
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Pfizer Acknowledges the Death of a Young Boy Who Died One Year After Participating in its Phase II Clinical Trial to Treat DMD

Pfizer has recently paused its Phase III clinical trial that has been assessing the Duchenne muscular dystrophy candidate fordadistrogene movaparvovec. The Phase II DAYLIGHT trial (NCT05429372), enrolled ten boys ages…

Continue Reading Pfizer Acknowledges the Death of a Young Boy Who Died One Year After Participating in its Phase II Clinical Trial to Treat DMD
Rare Community Profiles: #RAREis: Increasing Medical Access and Equity for the Hispanic Community through The Akari Foundation’s Initiatives
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Rare Community Profiles: #RAREis: Increasing Medical Access and Equity for the Hispanic Community through The Akari Foundation’s Initiatives

  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, advocates, scientists, and more.…

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CureDuchenne’s FUTURES National Conference

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FUTURES National Conference May 23-26, 2024 Orlando, FL The CureDuchenne FUTURES National Conference is an annual event focused on bringing education, connection, and hope to the entire Duchenne community.  Get…

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Gene Therapy Led to “Robust Microdystrophin Expression” in Duchenne Muscular Dystrophy Patient
source: pixabay.com

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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CureDuchenne’s Cares Dinner Session: Tampa, FL

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CureDuchenne Cares Dinner Session March 16, 2024 Tampa, FL A NEIGHBORHOOD DINNER FOR CAREGIVERS AFFECTED BY DUCHENNE AND BECKER JOIN US FOR A CASUAL THREE-HOUR DINNER ON SATURDAY EVENING TO…

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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: Baltimore, MD

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CureDuchenne Cares Workshop March 9, 2024 Baltimore, MD 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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