Rocket Pharmaceuticals, Inc.'s innovative therapies show promise after FDA setbacks. With potential $1B+ markets, RCKT offers ...

We designed our dream home in Mexico for $270,000. It was cheaper than buying in the US but came with extra costs we didn't ...

One in 10 Americans live with a rare disease, which often lacks effective treatments. Such is the case for PIGA-CDG, an ultra ...

Think acid reflux is harmless? Your heartburn could be hiding a deadly cancer secret. Here are the symptoms you should never ignore!

Travere’s stock price climbed by 14% following the announcement that it will seek an FDA label expansion for Filspari.

Tuesday, the agency signed off on SpringWorks’ Gomekli (mirdametinib) as a treatment for the rare genetic disorder ...

Rare diseases have become an attractive target for pharmaceutical companies both on the ASX and internationally.

Astrazeneca Pharma India advanced 1.21% to Rs 7557.05 after the company's standalone net profit surged to Rs 54.68 crore in Q3 FY25 as compared with Rs 15.80 crore in Q3 FY24.

A surprising new study suggests that COVID-19 may not have originated from bats or pangolins, but rather from a rare fusion of human diseases. Using an advanced AI-driven approach called max-logistic ...

Charleigh Pollock, 9, is being treated for a terminal neurological disorder known as Batten disease with twice-monthly infusions of a drug that costs about $1 million per year.

TEXARKANA, Texas - A Texarkana, Texas boy battling a rare genetic disease is preparing to head to Arkansas Children's ...

GOMEKLI™ is an oral, small molecule MEK inhibitor approved in the United States for the treatment of adult and pediatric ...