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 ...

We must understand that while rare diseases, by definition, affect a small portion of the population, they collectively ...

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, ...

Zolgensma gene therapy is the most effective in infants with SMA who are presymptomatic and 6 weeks old or younger, according ...