Αποτελέσματα Αναζήτησης
9 Οκτ 2024 · Duchenne muscular dystrophy (DMD) is a rare, X-linked neuromuscular disease caused by pathogenic variants in the DMD gene that result in the absence of functional dystrophin, beginning at...
- AAV gene therapy for Duchenne Muscular Dystrophy: lessons ... - Nature
In June 2023, FDA granted accelerated approval to...
- FDA approves first gene therapy for Duchenne muscular dystrophy ...
The FDA has granted accelerated approval to Sarepta’s gene...
- AAV gene therapy for Duchenne Muscular Dystrophy: lessons ... - Nature
Κυριότερες Ειδήσεις
23 Οκτ 2024 · In June 2023, FDA granted accelerated approval to delandistrogene moxeparvovec (Elevidys®), an adeno-associated virus (AAV) gene therapy for Duchenne muscular dystrophy (DMD) for boys aged 4–5 ...
23 Οκτ 2024 · A new single-dose intravenous gene therapy, delandistrogene moxeparvovec, creates “micro-dystrophin” — a scaled-down version of the large gene that encodes dystrophin — and targets the skeletal and heart muscles. A trial showed statistically significant improvement in the expression of dystrophin and improvement in motor function in 4 ...
23 Ιουν 2023 · The FDA has granted accelerated approval to Sarepta’s gene therapy delandistrogene moxeparvovec (Elevidys) for Duchenne muscular dystrophy (DMD). It is the first time the FDA has greenlit a...
20 Ιουν 2024 · Today, the U.S. Food and Drug Administration expanded the approval of Elevidys (delandistrogene moxeparvovec-rokl), a gene therapy for the treatment of Duchenne muscular dystrophy (DMD) for...
23 Ιουν 2023 · Today, the U.S. Food and Drug Administration approved Elevidys, the first gene therapy for the treatment of pediatric patients 4 through 5 years of age with Duchenne muscular dystrophy (DMD)...
Investment in gene therapy has led to the approval of exon skipping by regulatory agencies, multiple clinical trials of systemic microdystrophin therapy using adeno-associated virus vectors, and revolutionary genome editing therapy using the CRISPR technology.
