Gene Therapy SMA - Search News

GlobalData on MSN

Biogen’s salanersen gains FDA breakthrough therapy designation for SMA

The FDA's decision follows data from a Phase Ib study on salanersen.

Zacks Investment Research on MSN

BIIB's next-gen SMA therapy Salanersen wins FDA's breakthrough tag

Biogen BIIB recently announced that the FDA has granted Breakthrough Therapy designation to Salanersen, an investigational ...

The American Journal of Managed Care

Splicing Modifiers and Gene Therapy for SMA

Panelists discuss how splice modifiers work by enhancing protein production from the SMN2 gene, with risdiplam (Evrysdi) being an oral daily medication and nusinersen (Spinraza) being administered ...

Biogen’s Salanersen Receives FDA Breakthrough Therapy Designation for Spinal Muscular Atrophy

Designation is supported by an exploratory analysis from the Phase 1b study showing that some children with SMA previously treated with gene ...

The American Journal of Managed Care

STEER Data Open Door to SMA Gene Therapy for Wider Age Range of Children

The STEER study showed significant motor function improvement in children with SMA using intrathecal onasemnogene abeparvovec. The primary efficacy endpoint was met with a 2.39-point improvement in ...

Medscape

Gene Therapy Tops Nusinersen in Spinal Muscular Atrophy

Treatment with onasemnogene abeparvovec gene therapy for spinal muscular atrophy (SMA) type 1 was associated with less need for nutritional support or nighttime ventilation compared to recommended ...

MarketWatch

Novartis receives FDA approval for Itvisma(R), the only gene replacement therapy for children two years and older, teens, and adults with spinal muscular atrophy (SMA)

The MarketWatch News Department was not involved in the creation of this content. -- Itvisma (onasemnogene abeparvovec-brve) demonstrated improved motor function and stabilization in patients ...

Monthly Prescribing Reference

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Biogen’s salanersen gains FDA breakthrough therapy designation for SMA

BIIB's next-gen SMA therapy Salanersen wins FDA's breakthrough tag

Splicing Modifiers and Gene Therapy for SMA

Biogen’s Salanersen Receives FDA Breakthrough Therapy Designation for Spinal Muscular Atrophy

STEER Data Open Door to SMA Gene Therapy for Wider Age Range of Children

Gene Therapy Tops Nusinersen in Spinal Muscular Atrophy

Novartis receives FDA approval for Itvisma(R), the only gene replacement therapy for children two years and older, teens, and adults with spinal muscular atrophy (SMA)

One-Time Gene Therapy Itvisma Approved for Spinal Muscular Atrophy

Novartis’ Gene Therapy Shows Promise in Treating SMA

Gene therapy slows progression of spinal muscular atrophy type 2

Novartis' Gene Replacement Therapy For SMA Demonstrates Meaningful Efficacy And Safety Results