Spinal muscular atrophy (SMA) life expectancy varies between types. The most severe types of SMA have a life expectancy of less than 2 years, while less severe types do not impact life expectancy.

The phase 3 STEER study enrolled 127 treatment-naïve patients aged 2 to less than 18 years with SMA type 2 who were able to sit but had never walked independently.

A new registry-based study of more than 250 children with spinal muscular atrophy (SMA) types 2 and 3 shows that new disease-modifying therapies are having a meaningful impact on disease progression.

Novartis NVS reported promising new safety and efficacy results from its phase III studies of investigational intrathecal onasemnogene abeparvovec (OAV101 IT) in a broad group of patients aged 2 ...

Novartis announced the success of its Phase III STEER study for investigational intrathecal onasemnogene abeparvovec (OAV101 IT) in treating spinal muscular atrophy (SMA) Type 2 in children and ...

Babies with type 1 might also struggle to get the nutrients they need because they have problems swallowing and sucking. Life expectancy for babies diagnosed with type 1 SMA is usually 2 years or ...

Patricia Panzarino, or Pidgie, was born with spinal muscular atrophy type 2. She's a musician, and recent advances in treatment gave Pidgie the ability to record an album. This is Pidgie's story ...

Topline results from a phase 3 clinical trial showed an investigational gene therapy for young people with spinal muscular atrophy type 2 increased mobility and slowed disease progression ...

Different types of insulin work at different speeds in the body. This chart breaks down the types of insulin, their duration, and the different brands available.

The Gufrans are paying a high price to keep their children alive. Affan, seven, and Erhan, five, have Spinal Muscular Atrophy (SMA) - a rare genetic disorder that causes muscle deterioration and ...

Regina Philipps faced the challenges of a rare, devastating muscular disorder when her son Shane was diagnosed with spinal muscular atrophy (SMA) Type 2 at 10 months old. Each year, one in every ...

Novartis announced the success of its Phase III STEER study for investigational intrathecal onasemnogene abeparvovec (OAV101 IT) in treating spinal muscular atrophy (SMA) Type 2 in children and ...