FDA approves leucovorin for rare disorder

Digest more
FDA approves leucovorin—but not for autism, even after Trump admin officials touted drug last year
The Food and Drug Administration approved the drug leucovorin as a treatment for the rare genetic condition cerebral ​folate deficiency on Tuesday—but stopped short of approving it as a treatment for autism in general,

Continue reading

US FDA approves leucovorin for ultra-rare genetic disorder causing autism-like symptoms
FDA approves leucovorin for rare disorder, but not for autism after Trump's claims of drug's promise
FDA Finds Little Evidence That a Drug Touted by Trump Can Help People With Autism
The Food and Drug Administration on Tuesday approved a generic medication for a rare brain disorder, while walking back statements by President Donald Trump and other administration officials that the...

Continue reading

US FDA approves leucovorin for rare genetic disorder causing autism-like symptoms
FDA Approves Drug Trump Touted for Autism to Treat Other Neurological Disorder
Bait and Switch? RFK Jr.’s FDA Pivots on ‘Promising’ Autism Treatment Leucovorin
The FDA’s decision formally makes leucovorin the first approved treatment for people with cerebral folate deficiency caused by a harmful variant in the folate receptor 1 gene.

Continue reading

FDA approves leucovorin for rare genetic condition, but not for autism
FDA Officials Say Autism Claims for Drug Promoted by Donald Trump Lack Strong Evidence

Penn medical student who is genetic carrier of rare form of ALS on mission to develop gene therapy

Yentli Soto Albrecht, who is a genetic carrier of ALS, is working on research at Penn that could one day prove life-saving.
Earth.com

New FDA framework could expand treatment access for rare genetic diseases

The FDA proposes new rules allowing mutation-specific gene therapies to qualify for approval despite extremely small patient populations.
The Coast News

Carlsbad family shares son’s battle with ultra-rare disease

A Carlsbad family is sharing their son’s story after the 5-year-old was diagnosed with a rare genetic disorder and began a gene therapy trial.

Building Resilient Rare Disease Support in Malaysia with Pharm-D Health Science's VALENS Specialised Nutrition

As Malaysia commemorates National Rare Disease Day and marks the launch of the National Policy for Rare Diseases, long-term, sustainable care for individuals living with rare, complex genetic conditio
Rolling Out

The rare genetic mutation that prevents wrinkles

Meet the luckiest people on Earth — individuals carrying a rare genetic mutation that prevents wrinkles from forming even as they age well into their 80s. These genetic lottery winners have provided scientists with incredible insights into how aging ...

St. Louis Families Unite for Rare Disease Day 2026; Take Part Foundation Advances Critical Genetic Testing and Research

St. Louis Families Unite for Rare Disease Day 2026 as Take Part Foundation Advances Critical Genetic Testing and