Ionis Pharmaceuticals IONS signed a license agreement with Japan-based Ono Pharmaceutical. Per the terms, Ono has acquired ...

There was no treatment until Doctor Adrian Krainer with Cold Spring Harbor lab on Long Island led the way to creation of Spinraza, a first FDA-approved treatment for Spinal Muscular Atrophy.

In 2016, Spinraza became the first approved treatment for SMA, with exceptional results in infants with severe SMA – so exceptional, it was considered unethical to continue enrolling children ...

Spinraza is another example where the AB rating ... and available clinical data to effectively negotiate an appropriate price for their orphan drug. About the authors Bhavesh Patel is a principal ...