A study found Evrysdi (risdiplam) helps the body produce more SMN, and also revealed genetic differences between SMA types 2 and 3.

Patients treated with OAV101 IT showed an increase from baseline in HFMSE total score compared with sham control. Topline data were announced from a phase 3 trial evaluating intrathecal onasemnogene ...

Biomarkers for SMA were identified using LC-MS and machine learning, revealing differences in protein expression across SMA types. CNTN1 and NRXN3 proteins showed potential as prognostic biomarkers, ...

In a first-of-its-kind study, researchers compared the efficacy of preventative therapy for spinal muscular atrophy (SMA) between two well-matched study groups, using either gene therapy (onasemnogene ...

Fayaz Madoor conducted 45-day statewide padayatras to raise funds for a 2-year-old suffering from Spinal Muscular Atrophy ...

In a first-of-its-kind study, researchers compared the efficacy of preventative therapy for spinal muscular atrophy (SMA) between two well-matched study groups, using either gene therapy (onasemnogene ...

"Decreasing AMMO [active maximal mouth opening] over time is seen most often in patients with SMA type 2 with an estimated reduction of almost 1 mm per year...At this point, we propose that ...

A Rajasthan-based non-profit organisation has extended financial support of ₹1.21 crore to help fund the treatment of a young girl from Indore diagnosed with Spinal Muscular Atrophy (SMA) Type 2, a ...

Researchers evaluated oxygen saturation and arterial CO2 variables to screen for sleep disordered breathing in children with SMA type 1-3. Of the measures evaluated, only an oxygen desaturation index ...