Duchenne Muscular Dystrophy

The 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.

In a recent issue of JAMA, a team led by Benjamin N. Rome, MD, MPH, examined how the FDA’s accelerated approval process has moved 5 genetically targeted treatments for Duchenne muscular dystrophy (DMD) through its pipeline despite limited evidence on their efficacy.

Since 2016, 5 targeted treatments have received accelerated approval from the FDA for use in Duchenne muscular dystrophy (DMD), and the total spend for just 3 of them is $3.1 billion.

The 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.

Outside 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.

Data are scarce regarding the benefits of strength training for muscular dystrophies, for which there are no cures.

The Momentum trial from Sarepta Therapeutics is investigating SRP-5051 (vesleteplirsen) for use among male patients aged 8 to 21 years who have Duchenne muscular dystrophy amenable to exon 51 skipping.

The 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.

This 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.

MEDLINE, 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.

Patient-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.

Delandistrogene moxeparvovec-rokl (Elevidys; Sarepta Therapeutics) is currently approved to treat Duchenne muscular dystrophy in 4- and 5-year-old patients who have a confirmed DMD gene mutation.

A 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.

A 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.

This year’s top content in the Duchenne muscular dystrophy (DMD) space focused on a novel gene therapy approval, the high cost of care for this genetic disorder, and steps forward in other treatments.

Givinostat, 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.

Investigators 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.

Over 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.

The 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.

With improved survival becoming more common among individuals who have Duchenne muscular dystrophy (DMD), cardiomyopathy is also increasing in prevalence among this population.

This retrospective review compared appendicular lean mass (ALM) and ALM index among patients with Duchenne muscular dystrophy (DMD) or Becker muscular dystrophy (BMD).

The drug’s mode of action is different than other types of corticosteroids because it’s based on the differential effects on glucocorticoid and mineralocorticoid receptors.

This comprehensive comparative analysis examined the economic and health care resource utilization implications of initiating glucocorticoid and exon-skipping therapy for Duchenne muscular dystrophy (DMD).

RGX-202, a gene therapy for Duchenne muscular dystrophy, was well tolerated with no therapy-related serious adverse effects in 3 patients who received the level 1 dosage.

In 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.