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 ...

The first prenatal therapy for spinal muscular atrophy (SMA) showed promising ... It's one of three drugs approved for SMA, the others being nusinersen (Spinraza) and onasemnogene abeparvovec ...

Scientists report results from a promising new approach to treat the rare neurodegenerative disorder. Spinal muscular atrophy (SMA) is a progressive neurodegenerative disorder set in motion before ...

Prenatal therapy for spinal muscular atrophy (SMA) with risdiplam has ... to its main rival – Biogen's oligonucleotide Spinraza (nusinersen) – which has to be administered by injection into ...

These include Biogen’s once-quarterly IT administration of Spinraza (nusinersen ... the growing market in its recent report, Spinal Muscular Atrophy: Opportunity Assessment and Forecast ...

Novartis (NVS) announced positive safety and efficacy results from the Phase III program for investigational intrathecal onasemnogene abeparvovec, ...

Later efficacy of nusinersen treatment in adult patients with spinal muscular atrophy: A retrospective case study with a median 4-year follow-up. Improvement in a post-stroke pediatric patient ...

Spinal bulbar muscular atrophy (SBMA) causes a loss of motor neurons in the spinal cord and brainstem. It mainly affects facial and swallowing muscles and the muscles in the arms and legs.

Spinal muscular atrophy (SMA) is a debilitating genetic condition that’s usually fatal by a few years of age. But an intriguing case study might demonstrate a simple new treatment, with a child ...

marking a significant advancement in the treatment of spinal muscular atrophy (SMA), the leading genetic cause of infant death. Read Genentech’s press release here. This new formulation provides ...