New study findings are highlighting a common feature of type 2 spinal muscular atrophy (SMA), revealing that low body mass index (BMI)/age z-score is often seen in patients with the SMA subtype ...

Title: TOPAZ: A Phase 2 Study to Evaluate the Efficacy and Safety of Apitegromab (SRK-015) in Patients with Later-Onset Spinal Muscular Atrophy (Type 2 and Type 3 SMA): Topline Results ...

New 2-Year Data Show Genentech’s Evrysdi (risdiplam) Continues to Demonstrate Improvement or Maintenance of Motor Function in People Aged 2-25 With Type 2 or Type 3 Spinal Muscular Atrophy (SMA) ...

Spinal muscular atrophy with respiratory distress (SMARD) includes type 1 (SMARD1) and type 2 (SMARD2). Learn about symptoms, causes, treatments, and… READ MORE ...

People with type 2 and type 3 SMA are typically diagnosed after 6 months of age. Those with type 2 SMA are expected to achieve unassisted sitting, but never gain the ability to walk. Those with ...

The SUNFISH study is a 2 part, multicenter, randomized, double-blind, placebo-controlled phase 2/3 trial in patients aged 2-25 years with Types 2 or 3 SMA (N=231).

When I was born 63 years ago, doctors didn't know much about spinal muscular atrophy type 2, known as SMA2.My sister, who is 12 years older than me, had the same genetic condition, which often ...

The randomized, double-blind, sham-controlled phase 3 STEER study (ClinicalTrials.gov Identifier: NCT05089656) enrolled 127 treatment-naïve patients aged 2 to less than 18 years with SMA type 2 ...

In type 2 SMA, the onset of symptoms is between 6 months and 18 months. People with this condition may be able to sit at diagnosis but are likely to lose this ability over time. However, progressive ...