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Sarepta Announces Plans to Submit sNDAs for AMONDYS 45 and VYONDYS 53
Today, Sarepta Therapeutics announced plans to submit supplemental New Drug Applications (sNDAs) to the U.S. Food and Drug Administration (FDA) for AMONDYS 45 and VYONDYS 53, the company’s exon-skipping therapies for the treatment of individuals living with Duchenne amenable to skipping exons 45 or 53, respectively. Both therapies are currently approved under the FDA’s accelerated…
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Sarepta Therapeutics is preparing to submit supplemental new drug applications (sNDAs) to the FDA for converting the accelerated approvals of AMONDYS 45 and VYONDYS 53 to traditional approvals
Sarepta Therapeutics is preparing to submit supplemental new drug applications (sNDAs) to the FDA for converting the accelerated approvals of AMONDYS 45 and VYONDYS 53 to traditional approvals, supported by data from the ESSENCE confirmatory study combined with real-world evidence. Encouragingly, the FDA has confirmed acceptance of these data for the applications, with submission planned…
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The CureDuchenne Clinic Relocates in Greater Dallas to Meet Rising Demand for Specialized Neuromuscular Care
Expanded Facility Delivers Comprehensive, Multidisciplinary Care, Removing Barriers for Patients with Duchenne and Becker Muscular Dystrophy DALLAS – February 20, 2026 – CureDuchenne, a global leader in advancing research and improving patient care for individuals with Duchenne and Becker muscular dystrophy, today celebrated the relocation and expansion of The CureDuchenne Clinic within the Neurology &…
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MDA 2026: FDA decision on deramiocel for DMD expected by August
The latest Phase 3 trial results continue showing that deramiocel, an investigational cell therapy for heart disease related to Duchenne muscular dystrophy (DMD), significantly slows the progression of arm and heart damage in boys and men with DMD. Based on the positive Phase 3 results, Capricor Therapeutics has resubmitted an application for U.S. approval of…
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Living with Duchenne grants me unique insight into accessible design
For most of my life, Duchenne muscular dystrophy has defined how I navigate the world. Duchenne is a genetic condition that causes progressive muscle loss. Over time, it weakens the arms, legs, breathing muscles, and heart. Many adults with Duchenne, including me, use wheelchairs and rely on a ventilator to breathe. Daily life often involves…
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How to find a Duchenne specialist and certified care center
Riketa Smith, a certified medical assistant, shares resources families can use to identify Duchenne specialists and certified care centers. The post How to find a Duchenne specialist and certified care center appeared first on Muscular Dystrophy News.
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Advocating for your child at Duchenne doctor visits
Sheryl Marrazzo shares advice for fellow caregivers on preparing for medical appointments, advocating with facts, and communicating effectively with clinicians. The post Advocating for your child at Duchenne doctor visits appeared first on Muscular Dystrophy News.
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MDA 2026: Dyne therapy boosts strength, cognition in DM1
Myotonic dystrophy type 1 (DM1) patients in a Phase 1/2 clinical trial saw gains in motor function and cognitive measures after receiving Dyne Therapeutics’ zeleciment basivarsen (z-basivarsen), previously known as DYNE-101, supporting the recent initiation of a Phase 3 study. Findings from the Phase 1/2 ACHIEVE trial (NCT05481879) and the design of the HARMONIA Phase…
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Sarepta Announces Initiation of Screening and Enrollment for ENDEAVOR Cohort 8 in Non-Ambulatory Individuals Living with Duchenne
Sarepta Therapeutics, Inc. has announced screening and enrollment are underway in Cohort 8 of the company’s ENDEAVOR study. ENDEAVOR is an open-label, Phase 1b study assessing the expression and safety of ELEVIDYS in multiple cohorts of individuals living with Duchenne, and Cohort 8 aims to assess prophylactic sirolimus treatment as part of an enhanced safety…
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MDA 2026: Duvyzat shows DMD benefits even at lower dose levels
Treatment with Duvyzat (givinostat) was associated with functional gains in boys with Duchenne muscular dystrophy (DMD) regardless of the final dose they received, according to new analyses from a Phase 3 clinical trial. The treatment also led to a slower loss of functional muscle tissue and a slower replacement of muscle by fat on MRI analyses.…
