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

Based on a small trial with positive muscle performance data, Regenxbio is expected to pursue approval of its gene therapy ...

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

Karnataka launched free treatment for Spinal Muscular Atrophy (SMA) at the Indira Gandhi Institute of Child Health in ...

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

These updates will also be presented at the American Society of Gene and Cell Therapy and Muscular Dystrophy ... including Novartis' Zolgensma ® for children with spinal muscular atrophy. Designed to ...

MediciNova, Inc., a biopharmaceutical company traded on the NASDAQ Global Market (NASDAQ: MNOV) and the Standard Market of ...

Are you planning to get a baby? Protect your child by screening for hereditary disorders that you may pass to them ...

Following the game-changing success of Spinraza, a molecular-based therapy that was crucial in the treatment of SMA (Spinal Muscular Atrophy), which used to be ... genes and answer questions on ...

Zolgensma gene therapy is the most effective in infants with SMA who are presymptomatic and 6 weeks old or younger, according ...

We are working to raise funds for them through crowdfunding.” Zolgensma (onasemnogene abeparvovec-xioi) is a groundbreaking ...

What’s an example of a well-executed drug launch? Look no further than Reata’s commercialization of Skyclarys, the first ...