New data analysis suggests an investigational higher dose of SPINRAZA may lead to clinically meaningful improvements in motor functionA NURTURE study analysis shows 92 percent of children who initiated
Child at Royal Manchester Childrenâs Hospital is first in north to receive £1.79m life-saving gene-therapy drug
Zolgensma is a one-off gene therapy that treats Spinal Muscular Atrophy (SMA).
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Thank you for subscribingWe have more newslettersShow meSee ourprivacy notice A patient at Royal Manchester Childrenâs Hospital is the first in the north of England to receive a potentially life-saving drug on the
Texas doctors begin screening newborns for rare disease
Spinal Muscular Atrophy kills more infants than any other genetic condition, but San Antonio hospitals offer a miracle treatment. Author: Matt Houston (KENS) Updated: 10:52 PM CDT June 3, 2021
SAN ANTONIO Beginning this week, doctors will screen every newborn in Texas for a rare disease called Spinal Muscular Atrophy, or SMA.
Most children born with the disease lack the gene responsible for creating proteins the body requires to perform basic motor skills. Some infants with SMA cannot hold up their heads, and most are unable to walk.
About one in 10,000 kids are born with the disease. No genetic condition kills more infants than SMA.