Latest News
Gene Transfer Shows Promising Results for DMD
Novel Drug for DMD Will Undergo an Advisory Committee Review
Clinical Trial Assessing Novel Antisense Oligonucleotide for DMD Underway
DMD-Associated Myocardial Injury Treated With Steroids
CAP-1002 Shows Disease Modification in Patients With Later-Stage DMD
Aerobic Cycle Training May Improve Endurance in Boys With DMD
FDA Grants Orphan Drug Designation for Potential New DMD Therapeutic
New Model May Explain Variability in Duchenne Muscular Dystrophy Progression
Novel Marker for DMD May Be Superior to CK-M
Latest Clinical Trials
Insights
Both Objective and Subjective Measures Needed to Measure Disability in DMD
Both objective and subjective measures of disability in Duchenne muscular dystrophy can help physicians better care for their patients.
Life Aspirations Explored in Patients With DMD
It is unfortunate that the prevailing view on chronic illness is that it simply “happens” to someone, and that the best one can do is to cope with the difficulties attached, comply with treatment, and hope for the best. This is perhaps exacerbated by medical language; a patient is said to have been “diagnosed with”…
Corticosteroid Therapy Used to Treat DMD and More
Some therapeutic advancements are so central to the diseases they treat that they themselves define a specific era in the therapeutic history of the disease, even as physicians continue to search for a definitive cure. Some therapeutic discoveries have redefined and reinvigorated modern medicine as we know it. Take, for example, the discovery of penicillin,…
Patient Perspectives
Why the MDA Shamrock Is Nothing Short of Sensational
In the last 40-plus years, March has been a time of great significance for the Muscular Dystrophy Association (MDA).
NORD Eases Struggles of Rare Disease Patients and Caregivers
I’m grateful to have solid footing when it comes to the specialized level of care I’ve received for Duchenne muscular dystrophy (DMD).
From a Push Wheelchair to a High-Tech Power Ride
Our columnist, who has DMD, reflects on how wheelchairs have changed over the years.
Check out RDA’s DMD Gene Therapy Resource Center
DMD Features
Omidubicel Shows Promise as Replacement for Umbilical Cord Blood in Stem Cell Transplants
Gamida Cell is gearing up to begin sales of omidubicel from Israel to US hospitals for use in stem cell transplants, pending FDA approval of the therapy.
Ambitious NCATS Bespoke Gene Therapy Consortium Targets Ultrarare Diseases
The Bespoke Gene Therapy Consortium (BGTC) is an ambitious public-private partnership that aims to develop gene therapies for ultrarare diseases.
MDA Honors 2 Massachusetts Researchers for Groundbreaking Work in Duchenne
The Muscular Dystrophy Association (MDA) recently honored researchers Louis Kunkel, PhD, and Brenda Wong, MD, for their work in DMD.
New Mexico Single Mom’s Big Challenge: Raising Identical Twin Boys With Duchenne
Shannon Mosimann, a single mom in Albuquerque, New Mexico, talks about the challenges of raising identical twin boys with Duchenne muscular dystrophy.
DMD Conference Coverage
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CureDuchenne 2023 FUTURES Conference Registration Now Open
Registration is now open for the CureDuchenne 2023 FUTURES National Conference focused on Duchenne muscular dystrophy (DMD). The conference is “an annual event focused on bringing education, connection, and hope to the entire Duchenne community,” according to its sponsor, CureDuchenne. The conference will take place Friday, April 21 through Sunday, April 23, 2023, with a…
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