Roche and PTC Therapeutics' Evrysdi for spinal muscular atrophy (SMA) has been approved by the FDA for younger children with the rare disease, extending its use to include infants less than
Spinal muscular atrophy (SMA) therapy Spinraza has been a key earner for Biogen in recent years, but with sales now heading into reverse, the company is looking to extend its franchise.
Roche has highlighted new data with its oral treatment for spinal muscular atrophy (SMA) – Evrysdi – showing its benefits when given to pre-symptomatic babies with the rare disease.
Biogen is planning to trial its Spinraza spinal muscular atrophy (SMA) drug in patients who have not responded properly to Novartis' gene therapy Zolgensma, combining the two ultra-expensiv
Children in England with the rare muscle wasting disease spinal muscular atrophy (SMA) will get access to Biogen’s super-expensive new drug Spinraza after the manufacturer struck a deal wit
In an industry growing as quickly as cell therapy, keeping up with the demands of manufacturing and treatment is a challenge for the whole industry to tackle together, including stakeholder
Drug discovery is usually a goal-driven and iterative process, as researchers look for innovative new routes to target particular diseases and study those diseases in afflicted patients to learn ho