Genetically targeted technologies are built on Nobel Prize-winning science. They represent a scientific “quantum leap” that ...

Rare diseases - a medical condition so obscure that even your doctor has to Google. Despite their name, these diseases ...

Impilo's prototype platform scours medical records to pinpoint symptoms of unusual illnesses that are often overlooked by ...

Prince Frederik of Luxembourg died on March 1, 2025, at the age of 22 after a prolonged battle with POLG mitochondrial ...

The second annual CharlieFest fundraiser music event is in the works and slated to take place at Rodgers Theatre in Corning ...

PolG is one of many other rare disorders classified as mitochondrial ... Prince Frederik is "very representative of so many in our community that just ran out of time" while waiting for a cure ...

A rare disease in the US is one in 1,500, or fewer than 200,000 people, and an ultra-rare disease is one in 50,000 or fewer than than 6,000 people.

Prince Frederik of Luxembourg has died after a lifelong battle with a rare genetic disorder known as POLG mitochondrial ...

Still, Novo is steadfast in its goal to eventually become a leader in the space, the company’s EVP of rare disease, Ludovic ...

A young girl faces an uncertain future after she was diagnosed with an extremely rare and debilitating medical ... is very small for her age. The disease also affects her appetite.

ITF, IntraBio and Orchard are among the companies that have won FDA nods in the past year for Duchenne muscular dystrophy, ...

As we observe Rare Disease Day today, let us take a look at seven drugs poised to be approved to address rare diseases in ...