That’s a longstanding issue with rare-disease drug candidates, given the inherently small patient base available for trials. McCarthy said the panel’s input would be helpful as the FDA ...

The UK's regulator has granted early access to Alnylam's ultra-rare disease drug lumasiran, allowing patients with primary hyperoxaluria type 1 to gain access ahead of a regulatory decision ...

However, Tennessee’s Victor Sharrah sees monsters everywhere due to an ultra-rare condition called “demon face” syndrome that causes everyone to look like they have an evil smile ...

The reality star, 39, who starred in the 2016 series when it ... as I don’t know enough about it yet. Other than it’s rare and does have treatment options.' Charlotte then asked for those ...

The J.D. Power 100-Point Score is based on hundreds of thousands of independent and unbiased opinions of verified car owners. Measures the level of defects, malfunctions, and design flaws experienced ...

Editor’s Note: Call to Earth is a CNN editorial series committed to reporting on the environmental challenges facing our planet, together with the solutions. Rolex’s Perpetual Planet ...

Another special cosmic event is to occur this year, and it could be a "once-in-a-lifetime viewing opportunity," according to NASA. It’s a nova explosion located in a star system 3,000 light ...

It appears that one of the most limited Nike Air Max 1s to ever drop could be rereleasing soon. Official product images of ...

Main Camera Quad 50 MP, f/1.9 or f/4.0, 23mm (wide), 1.0"-type, 1.6µm, multi-directional PDAF, Laser AF, OIS 50 MP, f/1.8, 75mm (telephoto), 1/2.51", 0.7µm, dual ...

Opera GX is the gaming-focused version of the popular Opera web browser, and from what we have gathered, it is well-loved by the gaming community for what it offers. Still, there is a problem ...

Be delighted by the location and lifestyle on offer with this fully furnished, ground floor apartment featuring a security entrance, light-filled kitchen with brand new oven & cooktop, living ...