The SMA Victor

BIO101, also known as 20-hydroxyecdysone, is an investigational drug attracting interest in SMA, because it targets muscle through a pathway that i...

BIO101 is an investigational oral drug candidate attracting attention because it may approach Spinal Muscular Atrophy, from a very different direct...

Evrysdi, also known as risdiplam, has become one of the most widely available disease-modifying treatments for Spinal Muscular Atrophy, or SMA, but...

Some individuals with SMA may be able to hold a pen or pencil but become tired quickly, while others may struggle with grip strength, finger moveme...

Zolgensma, also known as onasemnogene abeparvovec-xioi, has transformed the treatment landscape for Spinal Muscular Atrophy, or SMA, but it is not ...

In simple terms, most people with SMA do not produce enough of a protein called SMN protein, which motor neurons need to survive and communicate wi...

SMA causes motor neurons to become damaged and eventually die when the body does not produce enough survival motor neuron, or SMN, protein. Once th...

Artificial intelligence is beginning to change how researchers study Spinal Muscular Atrophy, also known as SMA, with some of the newest 2026 resea...

Artificial intelligence is beginning to create new possibilities for people living with Spinal Muscular Atrophy, also known as SMA, but its greates...

In the past, many children were not diagnosed until weakness, delayed milestones, breathing problems, or other symptoms had already appeared. Today...

Three treatments that transformed the SMA landscape are Spinraza, Zolgensma, and Evrysdi, but each approaches the disease differently. Spinraza, al...

On November 24, 2025, the U.S. Food and Drug Administration approved Novartis’ Itvisma, also known as onasemnogene abeparvovec-brve, for adults and...