FDA Approves Oral Treatment for Spinal Muscular Atrophy
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The U.S. Food and Drug Administration approved Evrysdi (risdiplam) to treat patients two months of age and older with spinal muscular atrophy (SMA).

This is the second drug and the first oral drug approved to treat this disease.

~ SMA is a hereditary disease that causes weakness and muscle wasting because patients lose lower motor neurons that control movement.

~ Evrysdi contains a survival of motor neuron 2-directed RNA splicing modifier.

~ In two clinical studies, efficacy was established based on the ability to sit without support for at least five seconds and survival without permanent ventilation.

~ After 12 months of treatment, 41% of patients were able to sit independently for more than five seconds, a meaningful difference from the natural progression of the disease because almost all untreated infants with infantile-onset SMA cannot sit independently.

~ After 23 or more months of treatment, 81% of patients were alive without permanent ventilation, which is a noticeable improvement from typical disease progression without treatment.

~ Common side effects of Evrysdi include fever, diarrhoea, rash, ulcers of the mouth area, joint pain (arthralgia) and urinary tract infections.

~ The FDA granted this approval of Evrysdi to Genentech, Inc.

Source: https://www.fda.gov/news-events/press-announcements/fda-approves-oral-treatment-spinal-muscular-atrophy
Dr. M●●●●●●d I●●●●●l H●●●●n M●●●●●●a and 11 others like this8 shares
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