Intrathecal treatment with a vector-based gene therapy was associated with a greater improvement in motor function at 52 ...
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.
India Today on MSN2d
Suvendu Adhikari donates Rs 10 lakh for treatment of girl with rare spinal diseaseLeader of Opposition Suvendu Adhikari donated Rs 10 lakh from his salary towards the treatment of a girl from West Bengal's ...
Novartis' OAV101 IT gene therapy showed positive results in Phase 3 trials for SMA, with data supporting its potential as a treatment option.
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 ...
Phase III Trial Shows Novartis’ Gene Therapy Significantly Improves Spinal Muscular Atrophy Outcomes
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 ...
Spinal muscular atrophy (SMA) is a rare genetic disorder in which the onset of progressive neurodegeneration begins before birth, owing to loss of the protein SMN. Drugs such as risdiplam ...
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