The FDA has granted Breakthrough Therapy designation to radiprodil for the treatment of seizures associated with GRIN-related neurodevelopmental disorder with GoF mutations.

A true icon of custom cars, Winfield shared his knowledge with others through workshops, passing the flames to the next ...

When Bud Miller learned about the death of Gene Hackman earlier this week, he saw not just the death of a famous movie actor.

Cadillac’s Formula 1 team has received official approval to join the grid in 2026 and its owners insist the team will be ...

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Days after giving an impassioned remembrance of late actor Gene Hackman this past Sunday at the Academy Awards, it was ...

F or the first time ever, doctors have treated a rare genetic disease known as spinal muscular atrophy (SMA) in the womb – and the now 2-year-old child is showing no signs of the disease.

after becoming the first person to be treated for the motor-neuron condition while in the womb. The child’s mother took the gene-targeting drug during late pregnancy, and the child continues to ...

The daughter of beloved actor Gene Hackman says he and his wife ... “typical” for carbon monoxide poisoning. “When a car motor engine is left on…and it isn’t properly turned off ...

Gene therapies often require complicated procedures ... a high dose of the therapy improved motor function and quality of life in patients who received it. With this data in hand, Forbes said ...