Search

...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...

...The most common symptoms of spinal muscular atrophy (SMA) are muscle weakness and low muscle tone. Recognizing these and other symptoms early can help people get a diagnosis and start...

...People with spinal muscular atrophy (SMA) can develop long-term complications because of progressive muscle weakness. One complication that is especially common in Type 2 SMA is joint contractures. Contractures happen...

...Spinal muscular atrophy (SMA) must be diagnosed with a genetic test. Before suspecting SMA, doctors often use physical exams to help distinguish it from other similar neuromuscular disorders. A diverse...

...People with spinal muscular atrophy (SMA) often have difficulty breathing because of chest muscle weakness. Breathing support can make sure they get enough oxygen to meet their body’s needs. Breathing...

...Spinal muscular atrophy (SMA) is one of many disorders of muscles and the nerves that control them. These are called neuromuscular disorders. These disorders have similar symptoms but vary in...

...Spinal muscular atrophy (SMA) is a genetic disorder. This means that SMA is inherited by children from their parents, and it is present at birth. It is caused by mutations...

...Spinal muscular atrophy (SMA) is a genetic disorder that causes progressive (gets worse over time) muscle weakness. It is caused by a defect in the SMN1 gene, which is needed...