The SMA Victor

Zolgensma, is a gene replacement therapy designed to address the genetic root cause of SMA by delivering a new working copy of the SMN gene into th...

Evrysdi, also known as risdiplam, is an FDA-approved therapy for pediatric and adult patients with SMA, and it is a daily oral medication available...

Zolgensma, is a prescription gene replacement therapy approved in the United States for children under 2 years old with SMA caused by mutations in ...

Spinraza, also known as nusinersen, is a prescription medicine used to treat Spinal Muscular Atrophy in pediatric and adult patients, and it is giv...

Spinraza, also known as nusinersen, is a prescription medicine used to treat Spinal Muscular Atrophy in pediatric and adult patients. It is given t...

Spinal Muscular Atrophy, also known as SMA, is a rare genetic neuromuscular disease most commonly caused by changes in the survival motor neuron 1 ...

This gene gives the body instructions for making survival motor neuron protein, which is essential for the health of motor neurons. Motor neurons a...

One of the most important things to understand about SMA is that it often affects the muscles closest to the center of the body the hardest. Muscle...

SMA type can influence symptoms, age of onset, physical milestones, and the level of support a person may need. Traditionally, SMA has been describ...

For many years, SMA care focused mainly on managing symptoms and supporting quality of life, but the treatment landscape has changed significantly ...

Motor neurons are specialized nerve cells that send signals from the spinal cord and brainstem to the muscles, helping the body move, sit, stand, l...

SMA most commonly occurs when the SMN1 gene is missing or not working properly, which can lead to low levels of survival motor neuron protein. This...