Disease-Modifying Therapies in Spinal Muscular Atrophy and Duchenne Muscular Dystrophy

Key Points

• Improved clinical outcomes in patients following disease-modifying therapy, particularly noted in spinal muscular atrophy.
• Evidence shows that treatment leads to enhanced muscle function and strength based on multiple biomarkers.
• Observational analysis across multiple clinical centers highlights the effectiveness and safety of various therapies.
• These findings support the potential for broader implementation of therapies in managing progressive muscular diseases.