The efficacy of risdiplam in treating SMA was confirmed in a clinical trial conducted in 2020. The Food and Drug Administration approved the usage of this drug in SMA therapy for adults and children two months and older in August 2020.
FDA Approves Nusinersen Drug To Treat Spinal Muscular Atrophy
The United States Food and Drug Administration has recently approved Nusinersen drug to treat Spinal Muscular Atrophy.
The United States Food and Drug Administration has recently approved Nusinersen drug to treat Spinal Muscular Atrophy.
‘Risdiplam helped improve motor functions among children with
Spinal muscular atrophy (SMA). It also increased their life expectancy.’
In August 2020, the FDA approved the usage of risdiplam (Evrysdi) to treat SMA for adults and children two months and older. What Is Spinal Muscular Atrophy?
Spinal muscular atrophy (SMA) is a genetic disease affecting the central nervous system, peripheral nervous system, and voluntary muscle movement (skeletal muscle). It affects 1 per 8,000 to 10,000 people worldwide.
First Investigational Treatment For Spinal Muscular Atrophy Yields Positive Results In Clinical Trials
A major milestone was reached when nusinersen was shown to improve achievement of motor milestones in babies with infantile-onset spinal muscular atrophy.
A major milestone was reached when nusinersen was shown to improve achievement of motor milestones in babies with infantile-onset spinal muscular atrophy.
There is a gene called SM1 that produces SMN protein. When this gene is deleted or mutated, the person could not make enough SMN protein. This causes SMA as the brain fails to control voluntary movements.
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A gene called SMN2 can produce small amounts of SMN protein. Risdiplam is an oral medication which is capable of inducing SMN2 gene transcription. This helps the body to produce SMN protein even if the SMN1 gene causes issues.
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Test Your Knowledge on Spinal Muscular Atrophy (SMA)
Spinal muscular atrophy is a genetic disorder characterized by progressive loss of motor neurons in the spinal which control voluntary muscles. As a result, voluntary muscle activity such as walking, talking, breathing and swallowing becomes affected. Take this quiz to test your knowledge about spinal muscular atrophy.
Spinal muscular atrophy is a genetic disorder characterized by progressive loss of motor neurons in the spinal which control voluntary muscles. As a result, voluntary muscle activity such as walking, talking, breathing and swallowing becomes affected. Take this quiz to test your knowledge about spinal muscular atrophy.
In comparison with the participants who did not receive the drug, these children achieved other motor milestones. The life expectancy of these children also increased after risdiplam treatment.
“By the time an infant with SMA-1 comes to me, they have already lost 50 percent or more of their motor neurons. If we can find them before they become symptomatic, we can begin drug treatment very early and make a real difference for these children quite often by preventing the disease,” explained Darras, the co-founder of the Pediatric Neuromuscular Clinical Research Network (PNCRN) for SMA.
Source-Medindia
