Givinostat Receives Full FDA Approval for Duchenne Muscular Dystrophy
March 21st 2024The approval follows fast track, orphan drug, and rare pediatric designations and an original Prescription Drug User Fee Act date of December 21, which the FDA pushed back to March 21 to have more time to review Italfarmaco’s application.
Read More
Case Report Warns of Pneumothorax Risk in Duchenne Muscular Dystrophy
March 6th 2024The authors of this report stress the importance of awareness of COVID-19–related complications in young patients who have Duchenne muscular dystrophy, paying particular attention to a potential higher risk of respiratory infections in these patients.
Read More
Smartphone Tech Reveals Gait Differences in Duchenne Muscular Dystrophy
February 28th 2024Outside of a gait laboratory, this investigation compared gait pattern differences between children who have Duchenne muscular dystrophy (DMD) and their typically developing counterparts to gain more real-world data of DMD-associated gait characteristics.
Read More
Pre-, Postinfusion Protocol Recommendations for Delandistrogene Moxeparvovec Use in DMD
February 5th 2024The gene therapy delandistrogene moxeparvovec-rokl, also known as Elevidys (Sarepta Therapeutics) is indicated to treat Duchenne muscular dystrophy (DMD) in ambulatory patients aged 4 to 5 years, and Sarepta recently filed supplemental data with the FDA seeking to expand the labeled indication.
Read More
Translating DMD Clinical Trial Findings to Real-World Application Discouraging
January 31st 2024This analysis compared outcomes among patients who have Duchenne muscular dystrophy (DMD) and commercial insurance or Medicaid, focusing on how these differ between patients in clinical trials and those who receive treatment post approval.
Read More
DMD Guidance Could Benefit From Update, Experts Say
January 24th 2024MEDLINE, the Turning Research Into Practice database, Google Scholar, and organization guidelines were searched for their ongoing applicability to the management of Duchenne muscular dystrophy (DMD) and to determine if there is a need to develop new treatment recommendations.
Read More
Health State Utility Scores for DMD-Related Impairment Need to Better Reflect Patient Input
January 12th 2024Patient-reported outcomes data regarding measures of health-related quality of life, also known as health state utilities, in Duchenne muscular dystrophy (DMD) are scarce, with current knowledge in this area concentrated in caregiver feedback.
Read More
Proposed Guidance for DMD-Related Respiratory Function Evaluation in the UK
January 2nd 2024A multidisciplinary team has proposed respiratory care guidance for pediatric and adult patients living with Duchenne muscular dystrophy (DMD) in the United Kingdom (UK), with these recommendations meant to optimize DMD care day to day and in acute episodes.
Read More
Descriptions of Physical Limitations, Symptoms of DMD From the Patient, Caregiver Perspective
December 29th 2023A recent study encapsulates how patients and caregivers characterize the impact of Duchenne muscular dystrophy (DMD) on patients’ physical limitations and symptom burden, potentially helping to inform patient-centered strategies for assessing clinical outcomes in DMD research.
Read More
FDA Extends Review Time for Investigational Duchenne Agent Givinostat
December 15th 2023Givinostat, a histone deacetylase inhibitor from Italfarmaco, was supported by data from the phase 3 EPIDYS study, a randomized, double-blind, placebo-controlled, multicenter trial investigating the agent vs placebo.
Read More
New Investigation Analyzes Magnetic Resonance Measures of DMD for Disease Progression
December 7th 2023Investigators evaluated longitudinal MRI and spectroscopy outcomes and ambulatory function among 180 patients with Duchenne muscular dystrophy (DMD) to establish the utility and reproducibility of magnetic resonance measures of muscle quality at different disease stages.
Read More
EMBARK Data Support Delandistrogene Moxeparvovec for DMD Across All Study End Points
November 22nd 2023Over a 52-week treatment period, the gene therapy demonstrated robust evidence for a clinically meaningful benefit, including across several prespecified functional secondary end points vs placebo.
Read More
Data on Delandistrogene Moxeparvovec Can Inform Future Gene Therapies
November 17th 2023The FDA approved delandistrogene moxeparvovec-rokl (Elevidys, Sarepta Therapeutics) in June to treat Duchenne muscular dystrophy in ambulatory pediatric patients aged 4 through 5 who have a confirmed DMD gene mutation.
Read More
Exon-Skipping Therapy for DMD Linked to High Care Costs, Health Care Resource Utilization
October 25th 2023This comprehensive comparative analysis examined the economic and health care resource utilization implications of initiating glucocorticoid and exon-skipping therapy for Duchenne muscular dystrophy (DMD).
Read More
Case Report of Patient Death Following High-Dose Gene Therapy for DMD
October 19th 2023In this case study, a patient who had Duchenne muscular dystrophy (DMD) and received high-dose transgene therapy to upregulate cortical dystrophin subsequently developed acute respiratory distress syndrome and died.
Read More
Avidity Biosciences Gains FDA Orphan Drug Designation for Patients With DMD
October 9th 2023Sarah Boyce, president and CEO at Avidity Biosciences, discusses her leading role at the company, as well as antibody oligonucleotide conjugate drug, AOC 1044, currently in development for Duchenne muscular dystrophy (DMD) with mutations amenable to exon 44 skipping (DMD44).
Read More
FDA Approves Delandistrogene Moxeparvovec, First Gene Therapy to Treat Duchenne Muscular Dystrophy
June 22nd 2023The therapy, to be marketed as Elevidys, is approved for the treatment of ambulatory pediatric patients aged 4 through 5 years with DMD who have a confirmed mutation in the DMD gene.
Read More
Balancing Hopes for a Cure for SMA, DMD With Realistic Expectations
September 21st 2022A new study looks at the balancing act physicians and families must carry out when managing expectations at a time of great scientific advancement in spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD).
Read More