Healthline

FDA Doesn’t Endorse Leucovorin for Autism, OKs It for Rare Brain Disorder

The FDA declined to endorse leucovorin as a treatment for autism, but approved the drug to treat cerebral folate deficiency, ...

Viagra Ingredient Shows Promise for Rare Childhood Brain Disease in Surprising Study

Leigh syndrome is a rare metabolic disorder that begins in childhood and causes severe neurological and muscular problems. The disease has long been considered untreatable. In a pilot study involving ...

FDA approves leucovorin for rare disorder, but not for autism after Trump's claims of drug's promise

In September, Trump and the FDA commissioner announced the drug was under review to benefit patients with autism. The FDA didn't find evidence of that.

FDA Approves Drug for Rare Brain Disorder, Not Autism

But FDA officials said Monday that the agency later focused only on the strongest scientific evidence. That evidence only supported the drug’s use for people with the rare genet ...

4-Year-Old Girl with Rare Disorder Dies After Brain Injury Following Hospital Staff's Delay in Finding Medication

Family described Summer Mant as a "happy, adventurous, loving, cheeky, playful and very active child" ...

FDA OKs drug for rare brain disorder

The Food and Drug Administration on Tuesday approved a generic medication for a rare brain disorder, while walking back ...
American Songwriter

Daughter of Billy Joel Shares Hopeful Health Update Amid Brain Disorder Battle: “He’s Such a Trooper”

Alexa Ray Joel says her father, Billy Joel, is "doing great" and "a fighter" amid his battle with a rare brain disorder.

FDA clears repurposed GSK drug for ultra-rare brain disease instead of autism

Months after promising help for “hundreds of thousands of kids,” the FDA approved the decades-old medicine for a disease ...