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

Misdiagnosis or underdiagnosis of rare diseases in patients with diagnoses of common diseases can lead to delayed or inappropriate treatments, thereby complicating the management of both rare and ...

Earlier this year, researchers at Children's Hospital of Philadelphia and Penn created a first-of-its-kind drug customized to ...

GeneDx, which specializes in diagnostics for rare and ultra-rare genetic disorders, is helping to get kids the diagnostic attention they need as part of Diagnosis is Power. Currently, one in 10 ...

Rare diseases are defined as conditions affecting fewer than 200,000 people in the US or less than 1 in 2,000 in Europe. 1-2 While each disease is individually rare, collectively, they represent a ...

Median net sales for the 19 drugs in 2022 were $1.97 billion (range, $988 million-$8.36 billion). The drugs with the highest net sales were lenalidomide (Revlimid; $8.36 billion), ...

Rare Disease Day raises awareness for the thousands of rare diseases affecting millions. Advancements in technology, such as gene therapy, offer hope for potential cures for rare diseases in the ...

President Trump recently signed a wide-ranging executive order urging Congress to amend a crucial provision of a Biden-era law governing Medicare drug price negotiations, with the administration’s aim ...

Precigen believes that AI — combined with human interactions — is the future for rare disease marketing. The cell and gene therapy biotech has even tested out its hypothesis in its latest ad campaign.

VHL, a genetic cancer condition, recently was featured on "Grey's Anatomy." Feb. 10, 2011 — -- In a multi-episode arc on "Grey's Anatomy," actor Scott Foley plays a von Hippel-Lindau (VHL) ...