Clinical trialRSSToday
A grandmother shares how a small change in how her grandson's condition was measured helped him qualify for a Duchenne muscular dystrophy (DMD) treatment trial. Researchers adjusted the scoring system by 19 points, which gave him a second chance to participate in the study. This story highlights how trial eligibility rules can sometimes be adjusted to help more patients access new treatments.
WHY IT MATTERSIf your child with DMD has been told they don't qualify for a trial, this story shows that eligibility criteria can sometimes be reconsidered or adjusted—it's worth asking your doctor whether similar flexibility might apply to your situation.
ResearchRSSYesterday
A large study found that children with Duchenne muscular dystrophy (DMD) who started taking corticosteroids—a type of anti-inflammatory medicine—earlier in life had better muscle strength and movement than children who started the medicine about a year later. This shows that starting treatment as soon as possible after diagnosis helps preserve how well kids can walk, run, and use their muscles.
WHY IT MATTERSIf you have a young child with DMD, this research provides strong evidence to discuss with your doctor about starting corticosteroid treatment immediately after diagnosis rather than waiting, as earlier treatment may significantly slow muscle weakness.
NewsRSS2 days ago
Children with Duchenne muscular dystrophy (DMD) need physical therapy to stay mobile as long as possible, but doctors and families are trying to figure out how to balance therapy with letting kids just be kids and play. The article explores how to make treatment feel less like a chore and more like a normal part of childhood.
WHY IT MATTERSFamilies managing DMD face a real tension between following medical recommendations for physical therapy and allowing their children to enjoy normal childhood activities—this article addresses that specific struggle.
NewsRSS2 days ago
A tribute was published honoring Timothy Chan, a young man with Duchenne muscular dystrophy who recently passed away from a respiratory illness. The article celebrates how he inspired others through his approach to life, leadership, and compassion. Duchenne muscular dystrophy is a serious genetic condition that weakens muscles over time, and respiratory complications are a common concern for people with this disease.
WHY IT MATTERSThis story highlights the real-world impact of Duchenne muscular dystrophy and the importance of respiratory monitoring and care, which is critical for patients with this progressive condition.
NewsRSS2 days ago
A parent in rural Nebraska shares how their two sons with Duchenne muscular dystrophy (DMD) enjoy their summer break when school ends in mid-May. The article focuses on how the family adapts to seasonal changes and the boys' routines during warmer months. This is a personal story about managing life with DMD throughout the year.
WHY IT MATTERSParents of children with DMD can learn practical strategies for managing summer schedules and activities when school-based therapies and routines end.
NewsRSS2 days ago
Upsher-Smith Laboratories created two new websites to help people with Duchenne muscular dystrophy (DMD) who are using a medicine called Kymbee. One website is made for parents and caregivers of children with DMD and includes tips for talking to doctors and instructions for starting the treatment.
WHY IT MATTERSIf your child has DMD and is considering or starting Kymbee treatment, these new websites provide free resources to help you understand the medication and communicate with your healthcare team.
NewsRSS2 days ago
A person living with Duchenne muscular dystrophy (DMD) attended a performance of Les Misérables and reflected on how the musical's themes of second chances and perseverance mirror their own life journey with this progressive muscle disease. The article connects the emotional arc of the musical to personal experiences of living with a rare genetic condition. This is a personal perspective piece rather than medical news.
WHY IT MATTERSFor people with DMD, reading personal narratives from others living with the same condition can provide emotional support and help them feel less alone in their disease journey.
Clinical trialRSS2 days ago
The FDA has approved a clinical trial for a new cell therapy called remestemcel-L-rknd made by Mesoblast. The therapy will be tested in children ages 5-9 who have Duchenne muscular dystrophy (DMD), a serious muscle disease. About 76 children will participate in the trial and receive either the therapy or a placebo while continuing their regular DMD treatments.
WHY IT MATTERSThis trial represents a new treatment approach for DMD in young children who are still in a critical window for muscle development, offering families a potential alternative or complement to existing therapies.
AdvocacyRSS2 days ago
A mother of seven children shares her story about having three sons with Duchenne muscular dystrophy, a serious genetic muscle disease. She discusses her decision to have children despite knowing the genetic risks and explains why she doesn't regret her family planning choices. The article focuses on her perspective as both a caregiver and parent navigating life with this condition.
WHY IT MATTERSThis personal narrative highlights the lived experience and decision-making process for families with Duchenne muscular dystrophy, offering perspective on genetic counseling, family planning, and the emotional aspects of raising multiple children with the same rare disease.
NewsUNITERARE4 days ago
The PAN Foundation's financial assistance program for Duchenne muscular dystrophy patients is currently closed and not accepting new applications. This program previously helped patients pay for medications and treatment costs related to this serious muscle disease. You can check the PAN Foundation website to see when the program reopens or explore other financial assistance options.
WHY IT MATTERSIf you have Duchenne muscular dystrophy and need help paying for medications or treatments, you'll need to find alternative financial assistance programs since this particular fund is not currently accepting applications.