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...In 2016, Spinraza (nusinersen) became the first drug approved by the US Food and Drug Administration (FDA) to treat children and adults with spinal muscular atrophy (SMA). It works by...

...Having spinal muscular atrophy (SMA) does not mean it is impossible to date or have sex. Adults with SMA still have happy and intimate relationships. However, certain stereotypes about disabilities...

...Coughing is an important way to clear mucus and other secretions from our airways. This helps prevent the airways from getting blocked. Children with spinal muscular atrophy (SMA) have weaker...

...People with spinal muscular atrophy (SMA) have muscle weakness because of a defect in their motor neurons. In severe types of SMA, weakness can progress and cause serious complications. One...

...People with type 3 spinal muscular atrophy (SMA) have milder symptoms that progress slowly. They can stand and walk independently at some point but may lose the ability later in...

...Spinal muscular atrophy (SMA) is one of the most common genetic conditions that affect children. One in every 6,000 to 10,000 babies worldwide are born with SMA. Type 1 SMA...

...In 2019, the US Food and Drug Administration (FDA) approved Zolgensma (onasemnogene abeparvovec-xioi) to treat children under 2 years old with spinal muscular atrophy (SMA). Zolgensma is a gene therapy...

...In 2020, the US Food and Drug Administration (FDA) approved Evrysdi™ (risdiplam) to treat adults and children over 2 months old with spinal muscular atrophy (SMA). In 2022, that approval...

...People with spinal muscular atrophy (SMA) require ongoing, specialized care from a team of doctors and other health specialists. The team is usually managed by a doctor who specializes in...

...Treatment of spinal muscular atrophy (SMA) focuses on maintaining motor function, improving quality of life, and preventing complications. This usually includes physical therapy and nutritional support, as well as disease-modifying...

...People with spinal muscular atrophy (SMA) have progressive muscle weakness. Chest muscles that support breathing are often especially affected. This can lead to breathing problems because of undeveloped lungs, weak...

...Progressive weakness and wasting away of muscles. In SMA, it happens because of a genetic mutation leading to a loss of nerves that communicate with muscles. Inheritance pattern where the...

...Like many rare diseases, there are many misconceptions about spinal muscular atrophy (SMA). Most people are not familiar with SMA until a family or friend gets diagnosed with it. Experiences...

...As of 2024, the United States Food and Drug Administration (FDA) has approved 3 drugs to treat spinal muscular atrophy (SMA). They work in different ways to increase the amount...

...Children with spinal muscular atrophy (SMA) often have breathing problems that become worse during colds. This is because they cannot cough strongly enough to remove mucus from the lungs. This...

...Spinal muscular atrophy (SMA) is an inherited condition that can be diagnosed with a genetic test. People are usually diagnosed with SMA after they show symptoms unless there are cases...

...People with spinal muscular atrophy (SMA) can experience complications due to progressive muscle weakness. This includes joint and breathing problems. Children with SMA also seem to experience increased pain. We...

...Spinal muscular atrophy (SMA) is a group of inherited disorders. SMA causes certain muscles to become weak and waste away (atrophy). SMA may look different in every person. Historically, doctors...

...The life expectancy and prognosis of spinal muscular atrophy (SMA) vary widely between types. People with the most severe types may only live a few months. People with the mildest...

...Most of the common types of SMA start during childhood. Fewer than 5 percent of all SMA cases are type 4 SMA. This is the most common type of adult-onset...