A 6-week-old boy is fighting for his life inside OHSU Doernbecher's pediatric intensive care unit after being diagnosed with ...

Living with a rare disease often means years of uncertainty, misdiagnosis, and limited treatment options. But advances in ...

Blindness and deafness in some infants and sickle cell are among the conditions cured in recent gene therapy trials.

Zydus Lifesciences' wholly owned subsidiary Sentynl Therapeutics has entered into an agreement with Korea-based PRG S&T to license an investigational molecule, Progerinin (SLC-D011), ...

The world is experiencing a hidden epidemic with kidney disease. More than 850 million people worldwide have some form of ...

Stoke Therapeutics is developing a drug designed to reduce seizures and restore some of their lost developmental function.

Gaucher disease, a rare genetic disorder, often goes undiagnosed due to symptoms mimicking common ailments. Affecting the spleen, liver, and bones, it's also linked to an increased risk of Parkinson's ...

Biodexa Pharmaceuticals PLC (Nasdaq: BDRX), a clinical stage biopharmaceutical company developing a pipeline of innovative products for the treatment of rare diseases with unmet medical needs, today ...

The phrase "eye stroke" has recently appeared in news reports about a very rare side effect of weight-loss injections. It's ...

Mahanth Joishy recounts his battle with Sézary Syndrome, an extremely rare form of cancer that affects the skin, and how he ...

Many vaccines confer surprising knock-on, or indirect, health benefits when they have been put into widespread use.