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2 articles from the last 30 days matching "gene therapy"

🔴 BreakingDrug approvalRSS2 days ago

FDA Approves First-Ever Gene Therapy for Treatment of Genetic Hearing Loss Under National Priority Voucher Program

The FDA has approved Otarmeni, a new gene therapy treatment for genetic hearing loss. This is the first-of-its-kind treatment that uses a special virus to deliver healthy genes into the ear to help restore hearing. The approval was fast-tracked through a special FDA program that prioritizes treatments for serious diseases.

WHY IT MATTERSThis is the first gene therapy approved specifically for inherited hearing loss, offering a potential one-time treatment option for patients with certain genetic forms of deafness who previously had no curative options.
💬 Ask your doctorgenetic hearing losshereditary deafnessRead →
🔴 BreakingDrug approvalRSSApr 3

FDA Approves First Gene Therapy for Severe Leukocyte Adhesion Deficiency Type I

The FDA has approved a new treatment called Kresladi for a rare immune system disorder called Leukocyte Adhesion Deficiency Type I (LAD-I). This treatment works by using gene therapy, which means it fixes the faulty gene that causes the disease. This is the first gene therapy approved for this specific condition, giving patients with LAD-I a new treatment option.

WHY IT MATTERSPatients with severe LAD-I now have access to the first FDA-approved gene therapy for their condition, which could potentially offer a cure rather than just managing symptoms.
→ You can act on thisLeukocyte Adhesion Deficiency Type IRead →

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