Another milestone in Swiss science: Novartis put a treatment for deadly spinal atrophy on sale

[Deneb]

Swiss drugmaker Novartis has received US approval for its spinal muscular atrophy gene therapy Zolgensma. The Food and Drug Administration on Friday approved Zolgensma for children under the age of two with SMA, including those not yet showing symptoms. The approval covers babies with the deadliest form of the inherited disease as well as those with types where debilitating symptoms may set in later.
SMA is the leading genetic cause of death in infants. The disease often leads to paralysis, breathing difficulty and death within months for babies born with the most serious type 1 form. SMA affects about one in every 10,000 live births, with 50% to 70% having type 1.
The therapy uses a virus to provide a normal copy of the SMN1 gene to babies born with a defective gene and is delivered by infusion.
Novartis said it has so far treated more than 150 patients with Zolgensma. Its chief executive, Vas Narasimhan, described Zolgensma as a near-cure for SMA if delivered soon after birth. But data proving its durability extends to only about five years.
Novartis is expecting European and Japanese approval later this year. Zolgensma will compete with Biogen’s Spinraza, the first approved treatment for SMA.
Spinraza, approved in late 2016, requires infusion into the spinal canal every four months.

Why the Southern/Eastern European countries never get these scientific milestones? IMO, corruption and, as a result, lack of honesty, academic integrity and meritocracy.