FDA Unveils New Path for Approving Rare Disease Drugs for Just One Person

US regulators laid out new guidelines for approving custom-made treatments for individual patients, a move that could bring ...

Researchers to test gene therapy for rare diseases with a new FDA trial protocol

Earlier this year, researchers at Children's Hospital of Philadelphia and Penn created a first-of-its-kind drug customized to a unique genetic mutation to save an infant named Baby KJ from dying of a ...

Mom Learns Baby Is One of 6 Born with Rare Disease — and All 5 Others Are Dead. Now, She's 'Terrified' for the Future (Exclusive)

Amanda LaValle pushed for her daughter to get genetic testing. When she learns that her daughter is one of only six people in ...
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Her 5-year-old has a severe, rare disease. There's a cure, but she can't get it.

Catherine Illingworth, 39, knew something was wrong the moment her son's neurologist walked into the room. Illingworth requested the referral from her son's pediatrician based on instinct. George took ...
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FDA Announces Rare Disease Evidence Principles to Accelerate Treatments for Ultra-Rare Genetic Diseases

The U.S. Food and Drug Administration (FDA) announced Rare Disease Evidence Principles (RDEP) to allow for faster approval of treatments for ultra-rare genetic diseases. A product is eligible if it ...

Penn, CHOP will test gene therapy for rare diseases with new FDA trial protocol

Penn and CHOP researchers want to develop urea cycle disorder treatments using CRISPR gene-editing therapy. Get unlimited access to Inquirer.com and The Inquirer App, plus 5 articles each month to ...