Tenacious efforts at every level, from the individual clinician to the hospital to the state to Congress, will be needed to make sure patients can access life-saving gene therapies for neuromuscular diseases.
Posters presented at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference show that therapeutic advances in treating spinal muscular atrophy (SMA) are not uniformly making it into the hands of patients who could benefit.