Lisa, who lives with her family in Adelaide, Australia, began experiencing bouts of extreme fatigue about 18 years ago, and remembers being nearly asleep at odd hours of the day. Having two young children at the time, she shrugged it off as being a normal part of motherhood.

When Lisa’s fatigue worsened, she visited her doctor’s office. Her general practitioner diagnosed her with chronic fatigue syndrome, but Lisa still felt something was off and decided to get bloodwork done.

After Lisa’s blood tests revealed an abnormally high white blood cell count, she was referred to an …

  • Novartis Gene Therapies to initiate new pivotal confirmatory study to evaluate use of AVXS-101 intrathecal (IT) formulation in older patients with SMA to further support registration


Basel, September 23, 2020 Novartis Gene Therapies recently received feedback from the US Food and Drug Administration (FDA) following their review of data from the STRONG study of the intrathecal (IT) formulation of AVXS-101 in older …