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The FDA approves leucovorin for rare genetic condition and not for autism

Add Yahoo as a preferred source to see more of our stories on Google. The U.S. Food and Drug Administration on Tuesday approved leucovorin, a synthetic form of vitamin B9, as a treatment for a rare genetic condition that causes folate deficiencies in the brain

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.
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 ...

FDA approves leucovorin for ultra-rare genetic disorder causing autism-like symptoms

Washington — The U.S. FDA on Tuesday approved leucovorin, a decades-old generic drug, for use against an ultra-rare disorder called cerebral folate deficiency, but not for children with the much broader diagnosis of autism that it had promised last autumn to help, citing a lack of data.