AAV-Based Gene Therapy News, Researches and Articles

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We do not want children to die from a disease that can be cured. We want to give hope to families and ensure that all children with DMD are treated and regain their health.

Latest Articles

Sarepta Therapeutics Announces Approval in Japan of ELEVIDYS, a Gene Therapy to Treat Duchenne Muscular Dystrophy

Press Releases May 13, 2025 0

People aged 3 to under 8 who do not...

What’s Next for Patients with Duchenne Muscular Dystrophy (DMD) After Pharma Giants Pull Back from AAV-Based Gene Therapy Research?

Editorials May 13, 2025 0

Adeno-associated virus (AAV) vectors were once heralded as the...

Micro Dystrophin: 5 Critical Questions Everyone Is Thinking About

DMD May 10, 2025 0

Micro dystrophin and full-length dystrophin play crucial roles in...

Frequently Asked Questions About Elevidys Used for Duchenne Muscular Dystrophy

Approved Treatments December 26, 2024 5

A genetic condition known as Duchenne muscular dystrophy (DMD)...

Potential Upcoming New Gene Therapies for Duchenne Muscular Dystrophy

Research January 5, 2025 3

The results of new gene therapy studies for Duchenne...

What is Duvyzat (Givinostat)?

Approved Treatments October 13, 2024 1

For individuals with Duchenne muscular dystrophy (DMD), adults and...

AAV-Based Gene Therapy

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Which Mutations are Amenable to Exon 45 Skipping?

Entrada Therapeutics Receives Authorization in the United Kingdom to Initiate ELEVATE-45-201

Explore Of 5 Phases In Drug Development