Toddler 'in good spirits' after $2.9m gene therapy treatment
newsare.net
Little Devdan was diagnosed with spinal muscular atrophy (SMA) in 2019 when he was just a month old. But an infusion treatment last Saturday, thanks to a stunning campaign that raised more than $2.8 million, means the toddler may be well on his way to leadinToddler 'in good spirits' after $2.9m gene therapy treatment
Little Devdan was diagnosed with spinal muscular atrophy (SMA) in 2019 when he was just a month old. But an infusion treatment last Saturday, thanks to a stunning campaign that raised more than $2.8 million, means the toddler may be well on his way to leading a normal life. Last month, The New Paper reported that Devdan, who is now two years old, was set to be treated with Zolgensma, a onetime gene therapy for children with SMA. SMA is a neuromuscular disorder that affects the nerves and muscles and leads to muscle weakness that worsens over time. Devdan was confirmed to have Type 2 SMA when he was seven months old. Devdan can stand only when supported and is unable to walk or run. If left untreated, his muscles will degenerate and lose strength, and he could face total immobility in his teens. The treatment, widely touted as the most expensive drug in the world, was approved by the United States Food and Drug Administration (FDA) in 2019 and costs around $2.9 million. Read more