ST. LOUIS, Mo. (Ivanhoe Newswire) - Imagine your toddler learning to walk and talk and then all of a sudden, your child stops and starts regressing inside of themselves. That’s what happens to ...

The son of NBC News chief foreign correspondent Richard Engel, Henry Engel, died eight months ago at age 6 — but he’s still at the center of the research that will hopefully prevent other children ...

Scientists investigating the severe developmental disorder known as Rett syndrome have discovered a series of crucial molecular changes that occur long before symptoms appear. The findings could be ...

Rett syndrome is a rare neurodegenerative disorder that almost exclusively affects girls. It is typically diagnosed in babies between six and 18 months of age, when the first symptoms begin to appear.

The US Food and Drug Administration (FDA) has approved trofinetide oral solution (Daybue, Acadia Pharmaceuticals) as the first treatment of Rett syndrome in adults and children aged 2 years and older.

Please provide your email address to receive an email when new articles are posted on . Neurogene Inc. announced the FDA has granted clearance to its investigational new drug application for NGN-401, ...

In a comprehensive preclinical program, NGN-401 demonstrated robust therapeutic and safety benefits, delivering MECP2 to key brain regions affected by Rett syndrome NGN-401 utilizes Neurogene’s ...

Ella Frank, 15, has battled lifetime to challenges from rare neurological disorder. Her parents must replace special van to ...

The son of NBC News chief correspondent Richard Engel, Henry Engel died eight months ago at age 6 — but he's still at the center of the research that will hopefully prevent other children from losing ...