More research is needed to confirm gray matter, the darker tissue found in the brain and spinal cord, “as a potential disease ...

in treatment naïve patients with SMA type 2, aged two to less than 18 years who were able to sit, but never walked independently. Shares of Novartis have lost 3.6% in the past year aginst the ...

Application is supported by results from the DEVOTE study, which suggested that two doses of Spinraza (nusinerse) 50 mg taken ...

Novartis has revealed top-line results from the phase 3 STEER study, which involved treatment-naïve patients with SMA type 2 aged from two to 17 who can sit, but who have never walked independently.

in treating spinal muscular atrophy (SMA) Type 2 in children and young adults. The study demonstrated an increase in motor function, showcasing the therapy’s efficacy and safety compared to a ...

Intrathecal onasemnogene abeparvovec improved HFMSE scores in patients with SMA type 2, indicating enhanced motor function. The STEER study confirmed the therapy's favorable safety profile ...

Detailed results from the phase 3 SAPPHIRE trial, which involves patients with non-ambulatory type 2/3 SMA treated with Spinraza (nusinersen) or Evrysdi (risdiplam), are expected to be presented ...

Centre abdicates its constitutional obligation, criticises patients and experts on SMA issue: Our Bureau, New Delhi Friday, January 24, 2025, 18:15 Hrs [IST] Patients and public h ...

Health activists demand government action to reduce the cost of SMA medicines, citing constitutional obligations and ...

A woman with SMA type 3 became pregnant while on Spinraza treatment and gave birth to a healthy child, per a case report.