Efficient exon 44 skipping has been demonstrated in vitro, and intramuscular ... packaged together into a single AAV. This single AAV delivery system was able to rescue cardiac and skeletal muscle ...

Liver injury is a known complication risk of Elevidys and other gene therapies that use an adeno-associated (AAV) virus to deliver these one-time treatments to cells. That risk is already flagged ...

Elevidys is the only FDA-approved gene therapy for Duchenne muscular dystrophy (DMD) patients aged four and above. Liver damage is a known risk with Elevidys and other gene therapies that use ...

Sarepta Therapeutics said it will update its prescribing information for Elevidys® (delandistrogene moxeparvovec-rokl) after acknowledging the sudden death of a patient with Duchenne muscular ...

Giulio Cossu, MD, speaks to the lingering safety concerns related to ex vivo gene therapy in Duchenne muscular dystrophy (DMD ... through the use of an adeno-associated virus (AAV) vector.

Liver injury is a known possible side effect of the product, first approved by the U.S. FDA in June 2023 for DMD, as well as other AAV-mediated gene therapies, and the potential problem is highlighted ...

We inspect 10% of dentists in England each year. You can use our inspection reports to help you understand the quality of care. We do not give ratings to dentist services but we do highlight if a ...

As a result, higher-potency steroids should be avoided in children (Paller et al., 2005). Local adverse effects of topical steroids include striae, skin atrophy, telangiectasias, perioral ...

SAN FRANCISCO — Tecovirimat failed to reduce clade II mpox lesions, pain or viral clearance among participants in a study of the antiviral that was stopped early based on a futility analysis ...

High-potency corticosteroids should not be used in intertriginous areas because they can quickly cause skin atrophy, striae, and even ulceration. Weaker corticosteroids should be used on the face ...

DMD cardiomyopathy is a heart condition characterized by cardiac fibrosis, arrhythmias, and heart failure. Additionally, the FDA granted the BLA Priority Review with a Prescription Drug User Fee ...