Clinical trialRSSYesterday
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.
GrantRSSYesterday
The Muscular Dystrophy Association is holding its 26th annual fundraising gala in New York City on June 4 to raise money for ALS research. The event brings together financial leaders and medical researchers to support the search for treatments and cures for ALS, a disease that affects nerve cells controlling muscles.
WHY IT MATTERSFunding from this gala directly supports ALS research that could lead to new treatments, making it important for patients to know where research dollars are being invested and what progress is being made.
ResearchRSS2 days ago
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.
NewsRSS3 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.
NewsRSS3 days ago
A person who was diagnosed with limb-girdle muscular dystrophy 30 years ago finally took a genetic test to find out exactly which gene mutation caused their condition. Genetic testing can help doctors understand the specific type of muscular dystrophy a patient has, which may affect treatment options and family planning decisions. This story shows how genetic testing technology has improved over time and can provide answers even decades after an initial diagnosis.
WHY IT MATTERSGenetic confirmation of limb-girdle muscular dystrophy can unlock access to gene-specific clinical trials and emerging therapies that weren't available at the time of initial diagnosis.
NewsRSS3 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.
AdvocacyRSS3 days ago
A patient-led nonprofit called The Speak Foundation has created a network of specialized clinics called LGMD Centers of Excellence to help people with limb-girdle muscular dystrophy (LGMD), a rare muscle disease. This new network aims to solve a major problem: patients with LGMD have had trouble getting consistent care and doctors have struggled to develop new treatments because the patient population is very small and spread out.
WHY IT MATTERSThis coordinated clinic network could help LGMD patients access specialized care in one place and speed up the development of new treatments by making it easier for researchers to find and study patients.
NewsRSS3 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.
NewsRSS3 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.
NewsRSS3 days ago
A person with facioscapulohumeral muscular dystrophy (FSHD) is finding it harder to move around their home with a walker and is exploring other mobility options. They recently visited different places to look for solutions that might work better for their situation. This article discusses the process of finding new mobility devices when current ones stop working well enough.
WHY IT MATTERSPeople with FSHD often face progressive mobility challenges, and knowing what alternative devices exist can help maintain independence and quality of life as the disease progresses.
AdvocacyRSS3 days ago
Three organizations that work with facioscapulohumeral muscular dystrophy (FSHD) patients are joining together to make clinical trials better. FSHD is a rare muscle disease that causes weakness in the face, shoulders, and upper arms. This partnership wants to improve how these trials are designed so they can test new treatments more effectively.
WHY IT MATTERSBetter-designed clinical trials mean faster progress toward treatments for FSHD, and patient input through the FSHD Society ensures trials are structured in ways that actually work for people living with the disease.
NewsRSS3 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 trialRSS3 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.
AdvocacyRSS3 days ago
A person with a muscular dystrophy explains how using a wheelchair has improved their quality of life and ability to participate in daily activities. Rather than viewing the wheelchair as a symbol of disability, they see it as a tool that gives them more independence and function. The article challenges common misconceptions about wheelchairs and disability.
WHY IT MATTERSThis perspective piece helps patients with muscular dystrophy and other mobility conditions understand that mobility aids like wheelchairs can expand independence rather than limit it, which may help reduce stigma and encourage earlier adoption of helpful devices.
AdvocacyRSS3 days ago
When muscular dystrophy makes it hard to work, you may qualify for disability benefits that provide money and health insurance. This article explains how to navigate the process of getting these public benefits so you don't lose financial stability. Understanding your options can help you manage living with MD while keeping some independence.
WHY IT MATTERSPeople with muscular dystrophy who can no longer work need to know how to access disability benefits and medical coverage to avoid financial hardship during disease progression.
AdvocacyRSS3 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.
NewsUNITERARE5 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.
ResearchPUBMEDApr 17
Researchers in Saudi Arabia studied how different factors affect the stress and well-being of parents who have children with three rare genetic diseases: cystic fibrosis, congenital adrenal hyperplasia, and Duchenne muscular dystrophy. They surveyed 107 parents and found that things like whether parents work, family income, and cultural factors all play a role in how well parents cope with having a sick child. Understanding these factors can help doctors and support services better help families dealing with rare diseases.
WHY IT MATTERSIf you're a parent of a child with CF, CAH, or DMD, this research identifies specific factors affecting your quality of life—such as employment status and family support—that healthcare providers can now address to improve your family's well-being.
Clinical trialCLINICALTRIALSApr 15
Researchers are looking for 300 patients with Duchenne muscular dystrophy (DMD) to join a study about a medicine called givinostat. The study will track how safe the medicine is and how well it works for patients who are just starting to take it or have been taking it for less than 6 months. Patients will be followed for at least 2 years, with some being tracked for up to 5 years total.
WHY IT MATTERSThis real-world study will show how givinostat actually performs in everyday clinical practice for DMD patients in the US, providing practical safety and effectiveness data beyond what controlled trials reveal.
Clinical trialCLINICALTRIALSApr 9
Researchers at Columbia University are looking for 106 children and young adults with Duchenne Muscular Dystrophy (DMD) and Spinal Muscular Atrophy Type 3 (SMA Type 3) to join a study. The study will use special sensors and technology to track how people walk and move in their daily lives over a long period of time. The goal is to find new ways to measure how these diseases are progressing so doctors can better understand and treat them.
WHY IT MATTERSThis trial is now actively recruiting participants with DMD and SMA Type 3, offering a chance to contribute to developing better measurement tools that could help doctors track disease progression and evaluate future treatments more accurately.