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With FSHD, dealing with pain is a daily challenge for me
In a daily devotional I’m reading titled “Incurable Faith: 120 Devotions of Lasting Hope for Lingering Health Issues,” author Andrea Herzer describes pain as a thief, stealing her joy. Her words ring true for me as I navigate life with facioscapulohumeral muscular dystrophy (FSHD) and other health issues. Relentless discomfort is trying to steal my…
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Important Update: Sarepta Therapeutics Announces Strategic Restructuring and Pipeline Prioritization Plan to Maintain Long-term, Sustainable Growth and Provides Update on ELEVIDYS Label
CureDuchenne is sharing a community letter from Sarepta announcing a strategic company restructuring and pipeline prioritization plan. Additionally, Sarepta shared that the U.S. FDA has requested and Sarepta has agreed to include a black box warning in the ELEVIDYS label, resolving any material issues with the ambulant population indication. Sarepta has completed an Expert Committee on a protocol for the…
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First participant dosed in novel AAV-gene therapy trial for DMD-associated cardiomyopathy
Medera, and its clinical division Sardocor, announced that the first participant was dosed with AAV1.SERCA2a in a Phase 1b clinical trial for cardiomyopathy secondary to Duchenne. Cardiac complications are the leading cause of mortality in Duchenne, and this experimental gene therapy delivers the SERCA2a gene with the goal of correcting the calcium dysregulation in cardiac…
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MDA Ambassador Guest Blog: The Joy of Accessible Sailing
Mike Huddleston is 62 years old. He is originally from California and now lives in Maryland. Mike was diagnosed with spinal muscular atrophy (SMA) Type 3 at the age of 16. He started using a power wheelchair in 2015. He and his wife of over 40 years, Debbie, are both retired. Mike volunteers with several organizations…
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Capricor plans for deramiocel resubmission after FDA rejection
Capricor Therapeutics will resubmit its application for U.S. Food and Drug Administration (FDA) approval of its cell therapy deramiocel to treat Duchenne muscular dystrophy (DMD)-related heart disease, following the agency’s decision to not accept the original filing. According to a company press release, the FDA issued a complete response letter (CRL) requesting additional clinical data, stating…
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FDA indicated to Capricor that it will be unable to approve Deramiocel without additional data
Capricor Therapeutics announced that it has received a Complete Response Letter indicating that the FDA has completed the review of Deramiocel for Duchenne cardiomyopathy, but would be unable to approve the application in its current form. Capricor shared that the letter cites that their application does not meet the statutory requirement for substantial evidence of…
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Finding solace: Why I need July’s slower pace
July is my favorite month. Many people who know me might be surprised by that, as they’re aware that I’m a Christmas enthusiast who loves to decorate, making my house look like the North Pole for nearly eight weeks every winter. I spend the other 10 months of the year thrifting for the perfect Christmas…
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Capricor Therapeutics Receives Complete Response Letter Regarding Deramiocel (CAP-1002)
On July 9th, the U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) for Capricor’s Biologics License Application (BLA) for Deramiocel (CAP-1002), an investigational cell therapy for Duchenne cardiomyopathy. This decision means that the current application will not be approved in its present form, but it also presents a path forward by…
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Expedia’s New Initiative is Transforming Accessibility for Short-Term Rentals
Lorraine Woodward, founder and CEO of Becoming rentABLE. Expedia Group is not only opening the door, but widening the doorway, when it comes to accessible short-term rentals (STR) and vacation homes. As one of the largest travel companies in the world, hosting travel metasearch engines like Vrbo, Tavelocity, and more, the initiative to increase accessible…
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Post AAV Gene Therapy Resources
As therapeutic options for Duchenne advance, we are seeing continued development of additional AAV gene transfer therapies. There is currently one FDA approved gene therapy, ELEVIDYS, and several more in clinical trials. With all therapies, there are associated side effects and it’s critical that families and individuals who receive gene therapy (or any therapies) understand…
