After bringing Zolgensma to market in 2019 as the first gene therapy for spinal muscular atrophy, Novartis is back with an intrathecal formulation intended for older patients.
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 ...
To Novartis, fresh late-stage data support the idea that its intrathecal drug, which has the same active ingredient as ...
Novartis released two Phase 3 datasets on Wednesday that show a new version of its gene therapy Zolgensma led to motor ...
Gene therapy, with its offer of a possible cure for rare diseases like sickle cell, is losing early investors to ...
The FDA wants to end bulk production of copycat versions of weight loss drugs; telehealth companies and compounding ...
Sarepta Therapeutics (SRPT) stock is reeling this week following the death of a 16-year-old patient treated with its flagship gene therapy, ...
Delivery of onasemnogene abeparvovec into the intrathecal space was safe and effective for children with spinal muscular ...
Novartis’ Zolgensma gene therapy for spinal muscular atrophy is the most expensive drug in the world, but that hasn’t stopped it making a strong start in the US market. Zolgensma (onasemnogene ...
Gene therapy, which promises a possible cure for rare diseases like sickle cell, is losing early investors to higher-reward ...
Some results have been hidden because they may be inaccessible to you
Show inaccessible results
Feedback