FDA-approved gene therapies for Duchenne muscular dystrophy (DMD), such as Elevidys and exon-skipping drugs, aim to slow disease progression by producing functional forms of dystrophin or skipping harmful genetic segments.

Gene therapy strategies for DMD include altering genetic material or its usage, with several treatments approved or in development, though they are only suitable for specific mutations.

Genetic testing is essential to determine eligibility for gene therapies, as these treatments are limited to certain mutation types and age groups.

Ongoing clinical trials continue to evaluate the safety and effectiveness of new gene therapies, with some treatments still unapproved, and patients are encouraged to consult healthcare providers for participation and updates.

Gene editing technologies like CRISPR/Cas9 are still in research phases and not yet available outside clinical trials, aiming to directly correct genetic mutations in DMD.

Duchenne muscular dystrophy is a genetic neuromuscular disorder caused by mutations in the DMD gene, leading to progressive muscle weakness and potentially life-threatening complications.

Potential side effects of approved gene therapies include nausea, vomiting, fever, low platelets, and liver damage, while exon-skipping drugs may cause respiratory infections, headaches, allergic reactions, and other symptoms.

Current gene therapies only restore low levels of dystrophin and do not cure DMD, but they may help prolong mobility and delay disease symptoms.

Read-through therapy, exemplified by the FDA's acceptance of a new drug application for ataluren (Translarna) in October 2024, targets mutations causing premature stop codons to enable full-length dystrophin production.