
"The Power of Social Media": More than $2M crowdfunded for medicine to save a little girl's life
CBSN
Cairo — Roqaia Reda was born on July 13, 2020, in Alexandria, Egypt. She seemed a happy and healthy baby girl for her first six months, but then her parents started noticing some unusual symptoms. After a series of tests, Roqaia was diagnosed with spinal muscular atrophy (SMA), a rare degenerative neuromuscular disorder that leaves patients without control of their bodies.
In the most severe cases, which are generally those that manifest in early childhood, patients often die before they reach their second birthday. Most people with moderate cases live into adulthood, but they're often left unable to walk.
There are a couple drugs to treat SMA in young children, the most effective being a single-dose, intravenous treatment of Zolgensma. The gene therapy, which was only approved for use on young SMA patients in the United States in 2019, is often referred to as the most expensive drug in the world. Manufacturer Novartis puts the price tag for a treatment at about $2.1 million.

Mount Etna, the rambunctious volcano on the Italian island of Sicily, grumbled back to life on Monday, spewing hot ash and lava in a pyroclastic flow, the nation's volcano monitoring body said. There was no immediate report of any risk to the local population, which is accustomed to Etna's frequent eruptions, or to air travel.