Roche’s Evrysdi (risdiplam) is the market leader among the drugs for spinal muscular atrophy (SMA). Biogen’s Spinraza ...

Novartis is set to launch its one-off gene therapy Zolgensma for the ultra-rare muscle-wasting disease spinal muscular atrophy (SMA) in Germany at the start of July priced at 1,945,000 eu ...

REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will host a webcast to discuss the AFFINITY DUCHENNE® pivotal program and new clinical data, including the first functional data from the ongoing ...

Sriansh Ojha was fully into his "terrible twos" on an April afternoon at Cincinnati Children's as he squealed and chased his older sister around the sunlit lobby of the Schubert Research ...

Alpana Sharma, founder-director of CureSMA, a parent-led advocacy group, urges the government to include medicines for SMA in ...

The "Antisense & RNAi Therapeutics - A Global Market Overview" report has been added to ResearchAndMarkets.com's offering.The ...

CAR-T cell therapy Kymriah (tisagenlecleucel) for cancer, and spinal muscular atrophy gene therapy Zolgensma (onasemnogene abeparvovec). Viatris is accused of using the cells in the development of ...

A slow launch for Alzheimer's medicine Leqembi, a lackluster pipeline and a challenging drug launch environment are just a ...

With the success of SAPPHIRE, our Phase 3 registration study in spinal muscular atrophy and a successful financing ... they really don't define it by age short of gene therapy, which is approved under ...

SMA treatment Spinraza followed by Zolgensma improved motor function and prevented worsening of breathing difficulties in a ...

Muscle fibers in children with SMA type 1 show substantial differences in components of the myosin protein called heavy ...