-
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…
-
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.
-
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.
-
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…
-
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…
-
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.…
-
Sarepta trial enrolling for ELEVIDYS treatment with enhanced immunosuppression in non-ambulant individuals
Sarepta therapeutics is seeking to enroll 25 non-ambulant individuals with Duchenne in a clinical trial evaluating the use of sirolimus as part of an enhanced immunosuppressive regimen prior to and after Elevidys treatment. This data will aim to determine whether sirolimus treatment can help reduce the risk of acute liver injury with AAV gene therapy. …
-
MDA Ambassador Guest Blog: Navigating a Busy Life with a Feeding Tube
Richard Farrell Jr. is 19 years old and lives in Pennsylvania. He was diagnosed with Becker muscular dystrophy (BMD) when he was 5 years old. He loves to repair computers, play musical instruments, play video games, and talk with his friends. Richard “Richie” Farrell Jr. Living with a disability has required me and my parents…
-
MDA 2026: SGT-003 gene therapy shows early promise for DMD in trial
SGT-003, an investigational gene therapy for Duchenne muscular dystrophy (DMD), worked as expected to increase levels of microdystrophin — a version of the muscle-protecting protein that’s deficient in DMD — and preserve muscle health for boys in a clinical trial, according to new data. The one-time treatment was also well tolerated, avoiding the signs of liver…
-
Finding time for some quiet thoughts amid the travel and doctor visits
I’m one of those people who can get lost deep in thought. As a busy mom to many in a bustling household that could seem like chaos to others, my thoughts are often my only quiet time. I share seven children with my husband, Jason: Lexi, 24; Max, 20; Chance, 18; Rowen, 17; Charlie, 15;…
