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Snippets - A Small piece of News or Article

Snippets - A Small piece of News or Article

Meet the World’s most expensive drug: Zolgensma

The U.S. FDA recently approved the much talked about Novartis’s drug Zolgensma for treating a life-threatening condition Spinal Muscular Atrophy (SMA). SMA is a neuromuscular disorder that worsens the muscles over time. It happens due to a mutation in the SMN1 gene that results in the deficiency of an SMN protein, which is essential for the functioning and survival of motor neurons. SMA is a leading genetic cause of death in infants. With time the disease leads to difficulties in breathing, paralysis and even death of the babies born with Type 1- the most severe type of SMA. Zolgensma is a gene therapy that is designed to deliver a working SMN1 gene to facilitat...

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