Since 2016, the FDA has approved three disease-modifying treatments for spinal muscular atrophy, with several ...
A rare genetic condition, Spinal Muscular Atrophy (SMA) affects one in 7,000 people in India. It mostly affects children and ...
A Prescription Drug User Fee Act (PDUFA) target action date for apitegromab has been set for Sept. 22, 2025.
In the time since the Novartis gene therapy Zolgensma was approved for babies with spinal muscular atrophy, other treatments ...
Novartis' OAV101 IT gene therapy showed positive results in Phase 3 trials for SMA, with data supporting its potential as a ...
Delivery of onasemnogene abeparvovec into the intrathecal space was safe and effective for children with spinal muscular ...
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'We waited months for answers over our baby's condition - now he'll never be able to walk'“During the wait for the results, things deteriorated. He was choking constantly, not going to the toilet for days and his ...
Novartis also shared results from the open-label phase 3b STRENGTH study of OAV101 IT in SMA patients aged two to less than 18 years who had discontinued treatment with nusinersen or risdiplam, which ...
After bringing Zolgensma to market in 2019 as the first gene therapy for spinal muscular atrophy, Novartis is back with an intrathecal formulation intended for older patients.
Results from the Phase III STEER and Phase IIIb STRENGTH trials show that Novartis’ investigational intrathecal gene therapy onasemnogene abeparvovec ... in patients aged 2 to <18 years with spinal ...
Intrathecal onasemnogene abeparvovec (OAV101 IT), an investigational, one-time gene replacement therapy, demonstrated a ...
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