Set to enter larger trial across 5 countries, it works by permanently turning off gene that causes high bad cholesterol ...

Some patients with a rare form of ALS benefited from an experimental therapy, with biomarker evidence of reduced injury to neurons and even limited functional recovery.

Learn how a new genetic therapy could transform treatment for rare ALS—keep reading for remarkable patient outcomes.

EBC-129 is the first made-in-Singapore antibody-drug conjugate (ADC) to enter clinical development. It selectively targets a tumour-specific N-glycosylated epitope on both CEACAM5 and CEACAM6.

When Columbia neurologist and scientist Neil Shneider speaks to his ALS patients who volunteer for experimental therapies, he ...

Rocket Pharmaceuticals, Inc.’s (NASDAQ:RCKT) prospects have taken a significant hit on the company being forced to halt ...

Like opioids, SBI-810 works on the nervous system to relieve pain. The experimental compound is designed to target a receptor ...

Patients with some gastrointestinal tumors were able to be treated with immunotherapy drugs alone and avoid surgery, chemotherapy or radiation, the study found.

SB 535 formally paves the way for health care clinics to become 'experimental treatment centers' that can provide drugs still ...

An experimental drug known as ulefnersen has shown remarkable efficacy in treating a rare, aggressive form of ALS ...

Researchers say a full-scale clinical trial of the therapy could help many patients with a particularly aggressive form of ...

Novo Nordisk’s weight-loss drug Wegovy has been surpassed in weekly U.S. prescriptions by Eli Lilly’s Zepbound, and now the ...