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Rare Community Profiles: CureDuchenne Clinic in Greater Dallas Area Aims to Improve Treatment Accessibility and Care for Underserved Families with DMD and BMD

• Post author:Jessica Lynn
• Post published:May 10, 2023
• Post category:Becker muscular dystrophy/Duchenne Muscular Dystrophy

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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EDG-5506 Shows Benefits for Becker Muscular Dystrophy, Study Data Shows

• Post author:Jessica Lynn
• Post published:October 21, 2022
• Post category:Becker muscular dystrophy

  According to an article from Marisa Wexler in Muscular Dystrophy News, promising study data is available from the Phase 1 ARCH study. Within the study, researchers evaluated EDG-5506, an…

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Clinical Trial of Vamorolone Receives Orphan Grant Funding

• Post author:Kendall Mason
• Post published:October 5, 2021
• Post category:Becker muscular dystrophy/Duchenne Muscular Dystrophy/Muscular Dystrophy

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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ICYMI: Givinostat Stops BMD Muscle Shrinkage, Study Shows

• Post author:Jessica Lynn
• Post published:August 9, 2021
• Post category:Becker muscular dystrophy/Muscular Dystrophy

For years, researchers have evaluated Italfarmaco's givinostat for patients with Duchenne muscular dystrophy (DMD). In fact, a Phase 2/3 clinical trial showed that long-term givinostat treatment helped slow respiratory decline…

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Doctors Listened and Duchenne Muscular Dystrophy Patients Benefited

• Post author:Rose Duesterwald
• Post published:March 23, 2021
• Post category:Becker muscular dystrophy/Duchenne Muscular Dystrophy

Emflaza (deflazacort), a corticosteroid, was originally approved by the FDA in 2017 to treat Duchenne muscular dystrophy (DMD) in patients five years or older. This approval was expanded in 2019…

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New Phase 1 Study for Becker Muscular Dystrophy Treatment

• Post author:Kendall Mason
• Post published:November 19, 2020
• Post category:Becker muscular dystrophy/Duchenne Muscular Dystrophy/Muscular Dystrophy

Edgewise Therapeutics has announced their plan to initiate a Phase 1 trial of EDG-5506, their small molecule therapy for Becker muscular dystrophy. It will take place at Worldwide Clinical Trials…

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COVID-19 Advice for Patients with Becker or Duchenne Muscular Dystrophy

• Post author:Jessica Lynn
• Post published:May 13, 2020
• Post category:Becker muscular dystrophy/COVID-19/Duchenne Muscular Dystrophy

With 4.15 million cases across the globe, COVID-19 is at the forefront of discussion. Knowledge about the viral pandemic continues to grow each and every day. Yet there are still…

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The Cure Rare Disease Foundation is Trying to Change the “One Size Fits All” Approach Toward Treatment

• Post author:Trudy Horsting
• Post published:May 28, 2019
• Post category:Becker muscular dystrophy/Canavan disease/Charcot-Marie-Tooth disease/Cystic Fibrosis/Duchenne Muscular Dystrophy/Limb-girdle muscular dystrophy type 2A/Rare Disease/Sickle Cell Disease/Spinal Muscular Atrophy

The Cure Rare Disease Foundation The Cure Rare Disease Foundation's mission is to increase collaborative efforts between researchers in order to accelerate the development of customized therapies for individuals living with…

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