DYNE-251 received FDA breakthrough therapy designation, showcasing its potential for patients with Duchenne muscular dystrophy (DMD).
FDA Recommends Sarepta Resume DMD Gene Therapy Shipments for Ambulatory Patients
Quantitative Muscle Ultrasound a Promising Noninvasive Biomarker for DMD
TAS-205 Misses Primary End Point in Phase 3 DMD Trial
Accommodations as DMD Progresses Incur Substantial Household Costs
DYNE-251 Granted FDA Breakthrough Therapy Designation for DMD
The Value of Newborn Screening for DMD: Barry Byrne, MD, PhD
Dr Migvis Monduy on the Most Promising Areas of DMD Research
Insurance Insights: Dr Jason Shafrin Estimates DMD Insurance Value
AI-Based Electrocardiogram Interpretation Detects LVSD in Muscular Dystrophy
DMD Gene Therapy Shows Clinical Benefits in 8- and 9-Year-Old Patients
Navigating Hope, Risk, and the Unknown in a Changing DMD Treatment Landscape
DMD Gene Therapy Shows Efficacy, Safety in Patients Treated at 2 Years Old
Delandistrogene Moxeparvovec Shows Long-Term Safety, Tolerability in Pooled Trial Data
Quantitative Magnetic Resonance Outcomes Suggest Gene Therapy Slows DMD Progression
Consensus Guidelines Aim to Optimize DMD Gene Therapy Delivery
At-Home DMD Video Assessment Found Valid, Reliable
Eteplirsen Treatment Linked to Slower Cardiac Decline in DMD
WVE-N531 Shows Functional Benefits, Muscle Damage Reversal in DMD
Real-World Study Suggests DMD Gene Therapy Is Safe, Effective
Delandistrogene Moxeparvovec Shows Long-Term Impacts at 3 and 5 Years in DMD
MRI Could Aid in Early Differential Diagnosis of DMD, BMD
Survey Explores Needs of Patients With DMD, BMD Transitioning to Adulthood
SGT-003 Gene Therapy Shows Potential in DMD
Adults With DMD More Than Twice as Likely to Experience Fractures
Ifetroban Improves Heart Function in DMD
EMBARK 2: Delandistrogene Moxeparvovec Shows Sustained Benefits in DMD
Prophylactic Cardiac Medication May Prolong Survival in DMD
Quantifying the Altruism Value for a Rare Pediatric Disease: Duchenne Muscular Dystrophy
Later Loss of Ambulation Linked With Better Heart Health in DMD
Study Highlights Trends, Emerging Innovations in DMD Research
MRI Biomarker May Signal Early Heart Damage in DMD
FDA Pauses PepGen’s DMD Drug Trial in the US
Top 5 Duchenne Muscular Dystrophy Content of 2024
Real-World Challenges in DMD PMO Treatment
Viltolarsen Slows DMD Progression in Small Study
FDA Accepts Resubmitted NDA for Ataluren in Nonsense Duchenne Muscular Dystrophy
Sarepta Halts Development, Testing of DMD Therapy SRP-5051
A Year of DMD Gene Therapy Trial Failures
