News Related to Gene-targeted Therapies

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DMD Care Expands With Multidisciplinary Management, Earlier Pathology Focus

Apr 08, 2026

Duchenne muscular dystrophy care is shifting toward broader multidisciplinary management and closer attention to early pathology, biomarkers, and treatment timing. Clinicians described an expanding treatment landscape and identified neurology, cardiology, pulmonary, and endocrinology as core priorities.

Orphan Drugs Market Projected to Reach $486.51 Billion by 2032

Mar 23, 2026

The global orphan drugs market is projected to grow from $223.76 billion in 2023 to $486.51 billion by 2032, with North America leading at 38% market share. Over 500 orphan drugs are approved with more than 800 candidates in clinical trials, driven by regulatory incentives and major pharmaceutical company investments in rare disease therapies.