A two-and-a-half-year-old girl shows no signs of a rare genetic disorder, after becoming the first person to be treated for ...

A toddler was successfully treated for a rare genetic disease, spinal muscular atrophy, after world-first in-womb therapy.

Learn about the promising advance in prenatal treatment of spinal muscular atrophy, introducing a potential approach to suppress symptoms of the disorder in infants.

An infant with a fatal genetic disease has survived past the age of 2 with no signs of the condition, thanks to treatment ...

Spinal muscle atrophyorspinal muscular atrophy(SMA) is a genetic disorder that can affect babies, children, and adults. A ...

or patients with 5q SMA with a bi-allelic mutation in SMN1 and up to three copies of the SMN2 gene. Zolgensma is the world’s most expensive drug, with a price tag of more than $2.1 million per ...

Zolgensma can be used in patients with inherited mutations affecting genes known as SMN1, who have either ... have up to 3 copies of another gene known as SMN2. Around 550-600 babies are ...

Dec. 10, 2024 — In a single IV injection, a gene therapy targeting cBIN1 can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the ...

Gene regulation refers to the mechanisms that act to induce or repress the expression of a gene. These include structural and chemical changes to the genetic material, binding of proteins to ...