Post surgery the patient had to undergo physiotherapy and his lifelong wish to sit straight without falling has come true. He now has a 'strong and straight' spine. This is one of the finest ...
Infants with spinal muscular atrophy (SMA) who are presymptomatic and given the gene therapy Zolgensma in the first six weeks of life have better motor, respiratory, and nutritional outcomes, ...
Perspectives of patients with spinal muscular atrophy (SMA) and their caregivers should be weighed before making decisions around SMA-enhancing treatments.