FDA approves Wellcovorin for ultra-rare disease. The Trump admin touted it as a treatment for autism

The Food and Drug Administration (FDA) on Tuesday granted expanded approval to Wellcovorin for the ultra-rare disease ...
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, ...

FDA approves leucovorin as first drug for rare genetic disorder, after touting it as autism treatment

The move comes months after the Trump administration touted leucovorin as a potential therapy for a broader group of patients with autism symptoms.

FDA approves leucovorin to treat rare cerebral folate deficiency

The Food and Drug Administration has approved expanded use of leucovorin calcium tablets for treating cerebral folate ...

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.
WTEN

Malta family spreading awareness about rare genetic disorder

Rian McCann is almost seven years old. Her parents, Danielle and Frank described her as kind, sassy, smart and above all, resilient. Rian was born with CDKL5 Deficiency Disorder — which prevents her ...
Verywell Health

What Vitamin B6 Actually Does for Your Body

Learn what vitamin B6 does in your body, including benefits for brain health, energy, immunity, heart health. Plus, the best food sources of this essential nutrient.
Canadian Medical Association Journal

Irreversible blindness induced by vitamin A deficiency in a child with avoidant/restrictive food intake disorder secondary to Noonan syndrome

Key points A 4-year-old boy presented to our urgent ophthalmology clinic for photophobia and persistent eye rubbing. He had been born at 39 weeks via Cesarean delivery, without complications. Prenatal ...

Ultragenyx's gene therapy study shows ammonia reduction in rare metabolic disorder

Ultragenyx Pharmaceutical Inc. RARE shares are down on Thursday as the company reported data from its Phase 3 study of DTX301, a gene therapy for ornithine transcarbamylase (OTC) deficiency. • ...
Nature

Arginase Deficiency and Urea Cycle Disorders

Arginase deficiency is a rare autosomal recessive metabolic disorder that disrupts the final step of the urea cycle, impairing the conversion of arginine into urea and ornithine. In affected ...
Yahoo

Breakthrough study finds deficiency of this common nutrient could contribute to Alzheimer’s

A deficiency of the metal lithium in the body could be a key factor contributing to the development of dementia in Alzherimer’s patients, a groundbreaking new study reveals. The decade-long research, ...
Nature

Sulfite Oxidase Deficiency and Related Metabolic Disorders

Sulfite oxidase deficiency is a rare, inherited neurometabolic disorder arising from impaired detoxification of sulfite, a by-product of sulfur‐containing amino acid catabolism. The condition is most ...