The SMA Victor

For many individuals with SMA, reliable transportation is not just a convenience — it is a lifeline to medical appointments, therapy, school, emplo...

When these motor neurons weaken or atrophy, the muscles receive fewer signals from the brain and spinal cord, which can cause progressive muscle we...

Evrysdi, also known as risdiplam, is a disease-modifying treatment taken once daily that helps the SMN2 gene produce more functional survival motor...

Zolgensma, also known as onasemnogene abeparvovec-xioi, changed the SMA treatment landscape by offering eligible children under 2 years old a one-t...

Spinraza, also known as nusinersen, was the first disease-modifying treatment approved for SMA in the United States, but its price also makes it on...

One especially rare form is Spinal Muscular Atrophy with Respiratory Distress Type 1, also known as SMARD1, which is caused by disease-causing vari...

One critical fact every SMA patient should know is that Spinraza, also known as nusinersen, is a disease-modifying treatment approved for both pedi...

For many individuals with SMA, medical and adaptive equipment is not simply convenient — it can be essential for safety, health, communication, mov...

While new medications often receive the most attention, technology is also quietly changing what life with SMA can look like. Power wheelchairs, ad...

A major new development is giving the SMA community fresh hope: on September 11, 2026, the U.S. Food and Drug Administration approved Isembyld, als...

Current disease-modifying treatments have dramatically changed the natural history of SMA, but researchers are still searching for approaches that ...

Because SMA is considered rare, many people outside the disability and rare disease community may know very little about the condition or the daily...